Friday, March 31, 2017

Effective Management of Acute Intermittent Porphyria: Part 3




Effective Management of Acute Intermittent Porphyria:
Patients Deserve Access to Appropriate Care

What is Acute Intermittent Porphyria (AIP)?
AIP is one of a group of rare, inherited disorders called porphyrias that all involve the
overproduction and buildup of chemicals called porphyrins or porphyrin precursors. AIP is

Patients Diagnosed with AIP Deserve Access to Treatment
For people living with Acute Intermittent Porphyria (AIP), there are treatments available, but many
still encounter barriers to receiving accessible care at their preferred hospitals.

The American Porphyria Foundation (APF) urges all healthcare institutions to provide timely access
to medically necessary care. We encourage everyone involved in the care of patients with AIP to
demand timely access to FDA-approved treatments. We believe:

Regulators should advocate for patient access to approved treatments
Providers need to understand AIP and provide prompt, appropriate treatment Patients deserve access
and coverage for available, life-saving treatment  options
deaminase) and is characterized by acute attacks, often triggered by environmental factors or
hormone changes. In some cases, the cause

Signs and Symptoms

Gastrointestinal        
 Neurologic          
Cardiovascular            
 Urinary

Living with AIP

Because the symptoms of AIP mimic other common conditions, the diagnostic process can be long
suffering for years before receiving a proper diagnosis.
• Severe, intense abdominal pain
• Nausea and vomiting
• Constipation
• Diarrhea
• Pain in the extremities, back, chest, neck,or head
•  Muscle weakness
• Convulsions
• Mental symptoms
• Respiratory paralysis
• Fast heart beat
• High blood pressure
•Dark reddish or purple urine


This educational material is provided by the American Porphyria Foundation.
For more information,visit porphyriafoundation.com

Managing AIP
AIP is a genetic disease and there is no cure. However, there are strategies that can be used to
prevent the onset or severity of attacks:

•  Avoiding dieting or fasting, even for short periods
•  Avoiding certain medications that can trigger an attack
•  Limiting physical and emotional stress
•  Avoiding alcohol

People with frequent attacks can also consider wearing a medical alert bracelet.

References
1.  Anderson KE, Bloomer JR, Bonkovsky HL, et al. Recommendations for
the diagnosis and treatment of the acute porphyrias. Ann Intern Med. 2005;142:439-450.

2.   Bonkovsky HL, et al. Acute porphyrias in the USA: features of 108 subjects from porphyrias
consortium. The Amer. Jrnl of Med. 2014;127: 1233-1241.

3.   Crimlisk HL. The little imitator-porphyria: a neuropsychiatric disorder. J Neurol Neurosurg
Psychiatry. 1997;62:319-328.



Prompt Treatment is Critical
Even with proper diet and control of environmental factors, some patients still have recurring, and
often severe attacks. Acute attacks typically require hospitalization and delays in treatment can
be life-threatening and result in serious complications such as irreversible nerve damage.


Treatments can include:
•   Medication to manage pain and other symptoms
• Glucose and carbohydrates given orally or intravenously
•  Intravenous heme if the patient’s symptoms fail to improve within 36 hours of glucose treatment


Wednesday, March 29, 2017

What's all in the New Acute Porphyria Tool Kit? (AHP) Part 2

What's all in the New Acute Porphyria Tool Kit?  (AHP)

How can it benefit me or a caregiver?  How will it affect my Doctor's appointments?

All these tools come free of charge and can be downloaded any time.  

Please click on the link here: Access to Care Toolkit:  

Access to Care Toolkit for the Acute Porphyrias is now available


A downloadable Access to Care Toolkit is a resource designed to help patients living with Acute Intermittent Porphyria (AIP), Hereditary Coproporphyria (HCP), and Variegate Porphyria (VP) or their caregivers, loved ones and healthcare providers secure access to Panhematin at their preferred health facility.  We have recently learned of patients who are being denied this treatment from some hospitals and directed to secure another healthcare provider.  If this has happened to you or someone you know or care for, please use these tools to request help from your state and local representatives and health advocacy organizations.  We understand the debilitating effects of acute porphyria and hope these resources will help you secure access to Panhematin when you need it most.

The Toolkit contains the following materials:
*Healthcare Conversation Tracker is a simple form to record your conversations with doctors, insurance agents, etc.
*Customizable letter templates to record your details to use for doctors, state departments, insurance, etc.
*Access to Care Fact Sheet defines AIP, its symptoms and why it's important for patients to get immediate care
*Patient Bill of Rights can be used to support your appeal for access to treatment

This Toolkit can be found on the APF website.  Contact the APF office today if you have questions!
Part 2~




Effective Management of Acute Intermittent Porphyria:
Healthcare Conversation Tracker

Instructions
It is helpful to keep detailed records of your conversations and interactions with doctors, nurses, 
counselors, and any other healthcare staff. It is also important to keep track of your 
conversations with insurance providers. This information can support your appeals for access to care and treatment and should be provided with your letter.

Be sure to take notes on all your healthcare visits including the date, time, and outcomes, including any incidents or access to care issues. Also note any phone conversations. _____________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________


Be as much of the conversation, if possible. This will make it easier to remember. ____________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________


Also note conversations with 
your insurance provider and include the name of the representative. If you have to leave a telephone message, it is helpful to record the date, time, and person’s name.__________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________


This educational material is provided by the American Porphyria Foundation. For more information, 
visit porphyriafoundation.com


Use the form below to detail your issues with obtaining access to care and treatment for your condition, or reimbursement. Print multiple copies and record each visit or phone call you make. If you are unable to take notes, ask a relative, friend or caregiver if they can help. ___________________________________________________________________________________________________________________________________________________________________________________________________________________________

Submit a copy of this document along with other documents to support your claims. Remember to be thorough and capture as many details as possible.



Health Visit/Phone Call Report:_______________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________


Date:____________________________________________________________________                                                        
Time:____________________________________________________________________                                  
               

Address: ____________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________                                                                                          
                                                                                                    
I went in or called because:_____________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________                                                                        
                                                                                                    
I spoke to a (check one):         Doctor        Nurse        Counselor        Administrative Staff
His/Her name was:______________________________________________________________________________________________________________________________________________ 
I also interacted with a (check one):         Doctor        Nurse   
     Counselor        Administrative staff

I spoke with my insurance provider: __________________________________________________________________________________________________________________________________________________

The representative's name was: He/She told me:____________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________                                                          
                                                                                                    
Other Notes:_______________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________________                                                                                        
                                                                                                    

                                       




Monday, March 27, 2017

Access to Care Toolkit for the Acute Porphyrias is now available Part 1

What's all in the New Acute Porphyria Tool Kit?  (AHP)

How can it benefit me or a caregiver?  How will it affect my Doctor's appointments?

All these tools come free of charge and can be downloaded any time.  

Please click on the link here: Access to Care Toolkit:  

Access to Care Toolkit for the Acute Porphyrias is now available

A downloadable Access to Care Toolkit is a resource designed to help patients living with Acute Intermittent Porphyria (AIP), Hereditary Coproporphyria (HCP), and Variegate Porphyria (VP) or their caregivers, loved ones and healthcare providers secure access to Panhematin at their preferred health facility.  We have recently learned of patients who are being denied this treatment from some hospitals and directed to secure another healthcare provider.  If this has happened to you or someone you know or care for, please use these tools to request help from your state and local representatives and health advocacy organizations.  We understand the debilitating effects of acute porphyria and hope these resources will help you secure access to Panhematin when you need it most.

The Toolkit contains the following materials:
*Healthcare Conversation Tracker is a simple form to record your conversations with doctors, insurance agents, etc.
*Customizable letter templates to record your details to use for doctors, state departments, insurance, etc.
*Access to Care Fact Sheet defines AIP, its symptoms and why it's important for patients to get immediate care
*Patient Bill of Rights can be used to support your appeal for access to treatment

This Toolkit can be found on the APF website.  Contact the APF office today if you have questions!
Part 1~

Sample Letter from Patient
Note: The letter provided is only a sample providing suggestions to the writer for composing his/her own letter. It is the writer’s responsibility to detail his/her own thoughts and experiences in a personally acceptable manner. All content is ultimately the responsibility of the writer.

Dear [insert name],

I am a resident of [insert city or town] who lives with a serious medical condition known as Acute Intermittent Porphyria (AIP). AIP is a rare disease that is debilitating, painful and has profoundly impacted my life. I struggled for years to obtain a diagnosis. However, with my diagnosis, I was finally able to manage my disease with Panhematin, which I received at [insert name of hospital.]

I was devastated to learn that [insert name of hospital] will no longer honor my physician’s prescription and provide me with access to this treatment. Instead, I was told to find it through another hospital.

As a patient living with a rare disease, it is important I continue to receive timely access to the most effective treatment available. During an attack, I experience excruciating pain and [insert specific symptoms], that make it extremely difficult to find another hospital, much less get myself there.

Living with AIP is extremely challenging and recurrent, severe attacks have limited my ability to work and provide for myself and my family. It is extremely important that I have access to Panhematin at my preferred hospital. I worry that delays in treatment will result in a serious attack leading to hospitalization, and serious, potentially life-threatening complications.

I appeal to you to help me in urging [name of hospital] to provide continued access to the treatment I desperately need.

This is in keeping with my rights as a patient and in accordance with [insert name of state] Patient’s Bill of Rights and Responsibilities, which state:
  •  [insert exact language from bill of rights]
  • [insert exact language from bill of rights]

I appreciate your thoughtful consideration of my appeal and look forward to hearing from you soon.

Sincerely,

[Insert your name]




Sample Letter from Caregiver
Note: The letter provided is only a sample providing suggestions to the writer for composing his/her own letter. It is the writer’s responsibility to detail his/her own thoughts and experiences in a personally acceptable manner. All content is ultimately the responsibility of the writer.
                                                                     
Dear [name],

Imagine you, or a loved one, suffered from a potentially life-threatening rare disease only to have the hospital you know and trust deny access to the only treatment that helps.

Unfortunately, this is not a hypothetical story. This is my reality. I am a resident of [insert city or town] and I provide care to a person living with Acute Intermittent Porphyria (AIP), a rare, often debilitating and painful disease, that can lead to hospitalization if untreated.  He/she] has been receiving Panhematin, the only FDA approved hospital-based treatment for AIP for [insert number] years.

We were recently devastated to learn that [name of hospital] will no longer provide access to Panhematin. In fact, the [insert title of person you spoke with] turned us away and told us to find another hospital for treatment.

This is extremely troubling and disappointing, because it is critical that AIP patients have prompt, access to treatment. Delays can result in a severe, potentially life-threatening attack. Left untreated, an attack can result in serious complications such as irreversible nerve damage.

As a caregiver, the thought of watching my loved one suffer needlessly when there is a treatment available at their preferred hospital is unacceptable. I appeal to [insert organization/person receiving letter] to reconsider this misguided decision and continue to give patients the treatment they need and deserve in accordance with [insert name of state] Patient’s Bill of Rights and Responsibilities, which state:
  •  [insert exact language from bill of rights]
  • [insert exact language from bill of rights]

I appreciate your thoughtful consideration and look forward to hearing from you soon.

Sincerely,

[Insert your name]





Sample Letter from Healthcare Provider
Note: The letter provided is only a sample providing suggestions to the writer for composing his/her own letter. It is the writer’s responsibility to detail his/her own thoughts and experiences in a personally acceptable manner. All content is ultimately the responsibility of the writer.

Dear [insert name],

As the treating physician for a patient living with Acute Intermittent Porphyria (AIP), a rare disorder that often leads to extreme pain and debilitating symptoms, I was disappointed to learn that [name of hospital] will no longer provide [him/her] access to Panhematin, the only FDA-approved, hospital-based treatment for AIP attacks.

This is extremely troubling and I appeal to [insert organization/person receiving letter] to help ensure [name of hospital] provides continued access to this critical treatment in the interest of my patient’s health, and in keeping with [his/her] rights as a patient. Patients with AIP require prompt, unrestricted access to Panhematin that is prescribed under the care of physicians experienced in the management of porphyria, at hospitals that offer the necessary clinical and laboratory diagnostic and monitoring techniques. Delays in treatment can result in severe, potentially life-threatening attacks. Left untreated, attacks can lead to hospitalization, irreversible nerve damage and possibly death.

As healthcare providers to the community, [name of hospital] has an obligation to provide timely treatment to patients. Patients with AIP often experience excruciating pain, which greatly reduces their ability to manage changes in treatment schedules and locations. Turning my patient away from this facility is more than an inconvenience and could cause long-term health issues.

I appreciate your thoughtful consideration of my appeal and look forward to your reply.

Sincerely,

[Insert name]
[Insert title]





Friday, March 24, 2017

National Porphyria Awareness Week (NPAW) is right around the corner.

  

National Porphyria Awareness Week (NPAW) is right around the corner.
NPAW: April 22 - 29, 2017
See the story below as a great example of how to raise awareness in your community. 
For the second year in a row, the Cook family put on a fantastic benefit barrel race.  As you may know, the Cook brothers, Cason and Caul, have EPP and have set a great example about enhancing awareness of the disease   in their local area.  They have been hosting a Hat Day, supporting the APF and porphyria awareness, for many years in their hometown of Vernon, Texas.

There were over 159 entries for the race and an incredible outpouring of support from the community with volunteers and sponsors.
 Cook Family 1
Watch for stories like this from individuals and families who have changed the world of Porphyria Awareness in the community.

It is our hope that you will become involved in this year's awareness
activities.  
Let's RAISE PORPHYRIA AWARENESS!


Porphyria Awareness Week

"Remember....Research is the key to your cure!"

Wednesday, March 22, 2017

Access to Care Toolkit for the Acute Porphyrias is now available

Access to Care Toolkit for the Acute Porphyrias is now available

A downloadable Access to Care Toolkit is a resource designed to help patients living with Acute Intermittent Porphyria (AIP), Hereditary Coproporphyria (HCP), and Variegate Porphyria (VP) or their caregivers, loved ones and healthcare providers secure access to Panhematin at their preferred health facility.  We have recently learned of patients who are being denied this treatment from some hospitals and directed to secure another healthcare provider.  If this has happened to you or someone you know or care for, please use these tools to request help from your state and local representatives and health advocacy organizations.  We understand the debilitating effects of acute porphyria and hope these resources will help you secure access to Panhematin when you need it most.

The Toolkit contains the following materials:
*Healthcare Conversation Tracker is a simple form to record your conversations with doctors, insurance agents, etc.
*Customizable letter templates to record your details to use for doctors, state departments, insurance, etc.
*Access to Care Fact Sheet defines AIP, its symptoms and why it's important for patients to get immediate care
*Patient Bill of Rights can be used to support your appeal for access to treatment

This Toolkit can be found on the APF website.  Contact the APF office today if you have questions!
Access to Care Toolkit:  

Wednesday, March 15, 2017

Alnylam Receives European Medicines Agency PRIME Designation for Accelerated Assessment of Givosiran, an Investigational RNAi Therapeutic for the Treatment of Acute Hepatic Porphyrias

Alnylam Receives European Medicines Agency PRIME Designation for Accelerated Assessment of Givosiran, an Investigational RNAi Therapeutic for the Treatment of Acute Hepatic Porphyrias

03.01.2017

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading RNAi therapeutics company, announced today that the European Medicines Agency (EMA) has granted access to its Priority Medicines (PRIME) scheme for givosiran (ALN-AS1), an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyrias. The purpose of the PRIME initiative is to bring treatments to patients faster by enhancing the EMA's support for the development of medicines for diseases where there is an unmet medical need and where early clinical data show potential to benefit patients.
Promising results from the Phase 1 study of givosiran formed the basis of the application for PRIME. The ongoing Phase 1 trial is being conducted as a randomized, double-blind, placebo-controlled study. Specifically, data were recently reported in patients with acute intermittent porphyria (AIP) experiencing recurrent attacks. As presented at the 2016 American Society of Hematology (ASH) meeting, givosiran demonstrated initial evidence for clinical activity in AIP patients with meaningful reductions in the number and frequency of porphyria attacks. In the first two dose cohorts, givosiran was found to be generally well tolerated with no drug-related serious adverse events. In the third dose cohort, which remains blinded, one death due to acute pancreatitis, considered unlikely related to givosiran or placebo, was reported after the data transfer date.
"We are pleased to have givosiran accepted into the PRIME program. We believe givosiran could be a potentially transformative treatment option for patients with acute hepatic porphyrias, a family of debilitating and life threatening diseases with enormous unmet medical need," said Jeff Miller, Vice President, General Manager, Givosiran Program at Alnylam. "We look forward to collaborating with the EMA on the accelerated assessment of givosiran, with the goal of advancing this investigational medicine into a Phase 3 trial in late 2017."
Givosiran has previously been granted Orphan Drug Designations in both the EU and the U.S. for the treatment of acute hepatic porphyrias. Through the PRIME program Alnylam will have enhanced scientific and regulatory support from the EMA, including its advice on optimization of the development pathway and the potential for accelerated assessment of the Marketing Authorisation Application (MAA).
About Givosiran
Alnylam is developing givosiran (formerly known as ALN-AS1), a subcutaneously administered, investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyrias, including acute intermittent porphyria (AIP). AIP is an ultra-rare autosomal dominant disease caused by loss of function mutations in porphobilinogen deaminase (PBGD), an enzyme in the heme biosynthesis pathway that can result in accumulation of toxic heme intermediates, including aminolevulinic acid (ALA) and porphobilinogen (PBG). Patients with AIP can suffer from acute and/or recurrent life-threatening attacks characterized by severe abdominal pain, neuropathy (affecting the central, peripheral or autonomic nervous system), and neuropsychiatric manifestations. Givosiran is an ESC-GalNAc-siRNA conjugate targeting ALAS1, a liver-expressed, rate-limiting enzyme upstream of PBGD in the heme biosynthesis pathway. Inhibition of ALAS1 is known to reduce the accumulation of heme intermediates that cause the clinical manifestations of AIP. Givosiran has the potential to be a novel treatment approach for the prevention of recurrent attacks. Givosiran is an investigational compound, currently in early stage clinical development. The safety and efficacy of givosiran have not been evaluated by the U.S. Food and Drug Administration or any other health authority.
About Acute Hepatic Porphyrias
The porphyrias are a family of rare metabolic disorders with mostly autosomal dominant inheritance predominantly caused by a genetic mutation in one of the eight enzymes responsible for heme biosynthesis. Acute hepatic porphyrias (AHP) constitute a subset where the enzyme deficiency occurs within the liver, and includes acute intermittent porphyria (AIP), hereditary coproporphyria (HCP), and variegate porphyria (VP). Exposure of AHP patients to certain drugs, dieting, or hormonal changes can trigger strong induction of aminolevulinic acid synthase 1 (ALAS1), another enzyme in the heme biosynthesis pathway, which can lead to accumulation of neurotoxic heme intermediates that precipitate disease symptoms. Patients with AHP can suffer from a range of symptoms that, depending on the specific type, can include acute and/or recurrent life-threatening attacks with severe abdominal pain, peripheral and autonomic neuropathy, neuropsychiatric manifestations, cutaneous lesions and possibly paralysis and death if untreated or if there are delays in treatment. There are no approved treatments for the prevention of attacks; the only approved treatment for acute attacks is hemin for injection (Panhematin® or Normosang®), a preparation of heme derived from human blood. Hemin requires administration through a large vein or a central intravenous line and is associated with a number of complications including thrombophlebitis or coagulation abnormalities. Chronic administration of hemin may result in renal insufficiency, iron overload, systemic infections (due to the requirement for central venous access) and, in some instances, tachyphylaxis.
About GalNAc Conjugates and Enhanced Stabilization Chemistry (ESC)-GalNAc Conjugates
GalNAc-siRNA conjugates are a proprietary Alnylam delivery platform and are designed to achieve targeted delivery of RNAi therapeutics to hepatocytes through uptake by the asialoglycoprotein receptor. Alnylam's Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate technology enables subcutaneous dosing with increased potency and durability, and a wide therapeutic index. This delivery platform is being employed in nearly all of Alnylam's pipeline programs, including programs in clinical development.
About RNAi
RNAi (RNA interference) is a revolution in biology, representing a breakthrough in understanding how genes are turned on and off in cells, and a completely new approach to drug discovery and development. Its discovery has been heralded as "a major scientific breakthrough that happens once every decade or so," and represents one of the most promising and rapidly advancing frontiers in biology and drug discovery today which was awarded the 2006 Nobel Prize for Physiology or Medicine. RNAi is a natural process of gene silencing that occurs in organisms ranging from plants to mammals. By harnessing the natural biological process of RNAi occurring in our cells, the creation of a major new class of medicines, known as RNAi therapeutics, is on the horizon. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, target the cause of diseases by potently silencing specific mRNAs, thereby preventing disease-causing proteins from being made. RNAi therapeutics have the potential to treat disease and help patients in a fundamentally new way.
About Alnylam Pharmaceuticals
Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi. The company is leading the translation of RNAi as a new class of innovative medicines. Alnylam's pipeline of investigational RNAi therapeutics is focused in 3 Strategic Therapeutic Areas (STArs): Genetic Medicines, with a broad pipeline of RNAi therapeutics for the treatment of rare diseases; Cardio-Metabolic Disease, with a pipeline of RNAi therapeutics toward genetically validated, liver-expressed disease targets for unmet needs in cardiovascular and metabolic diseases; and Hepatic Infectious Disease, with a pipeline of RNAi therapeutics that address the major global health challenges of hepatic infectious diseases. In early 2015, Alnylam launched its "Alnylam 2020" guidance for the advancement and commercialization of RNAi therapeutics as a whole new class of innovative medicines. Specifically, by the end of 2020, Alnylam expects to achieve a company profile with 3 marketed products, 10 RNAi therapeutic clinical programs - including 4 in late stages of development - across its 3 STArs. The company's demonstrated commitment to RNAi therapeutics has enabled it to form major alliances with leading companies including Ionis, Novartis, Roche, Takeda, Merck, Monsanto, The Medicines Company, and Sanofi Genzyme. In addition, Alnylam holds an equity position in Regulus Therapeutics Inc., a company focused on discovery, development, and commercialization of microRNA therapeutics. Alnylam scientists and collaborators have published their research on RNAi therapeutics in over 200 peer-reviewed papers, including many in the world's top scientific journals such as Nature, Nature Medicine, Nature Biotechnology, Cell, New England Journal of Medicine, and The Lancet. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For more information about Alnylam's pipeline of investigational RNAi therapeutics, please visit www.alnylam.com.
Alnylam Forward Looking Statements
Various statements in this release concerning Alnylam's future expectations, plans and prospects, including without limitation, Alnylam's views with respect to the potential for RNAi therapeutics, including givosiran, its expectations regarding the timing of clinical studies, including the initiation of a Phase 3 trial for givosiran following interactions with regulatory authorities, its expectations regarding scientific and regulatory support for givosiran from the EMA and collaborating with the EMA on the accelerated assessment of givosiran, its expectations regarding its STAr pipeline growth strategy, and its "Alnylam 2020" guidance for the advancement and commercialization of RNAi therapeutics, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, Alnylam's ability to discover and develop novel drug candidates and delivery approaches, successfully demonstrate the efficacy and safety of its product candidates, the pre-clinical and clinical results for its product candidates, which may not be replicated or continue to occur in other subjects or in additional studies or otherwise support further development of product candidates for a specified indication or at all, actions or advice of regulatory agencies, which may affect the design, initiation, timing, continuation and/or progress of clinical trials or result in the need for additional pre-clinical and/or clinical testing, delays, interruptions or failures in the manufacture and supply of our product candidates, obtaining, maintaining and protecting intellectual property, Alnylam's ability to enforce its intellectual property rights against third parties and defend its patent portfolio against challenges from third parties, obtaining and maintaining regulatory approval, pricing and reimbursement for products, progress in establishing a commercial and ex-United States infrastructure, competition from others using technology similar to Alnylam's and others developing products for similar uses, Alnylam's ability to manage its growth and operating expenses, obtain additional funding to support its business activities, and establish and maintain strategic business alliances and new business initiatives, Alnylam's dependence on third parties for development, manufacture and distribution of products, the outcome of litigation, the risk of government investigations, and unexpected expenditures, as well as those risks more fully discussed in the "Risk Factors" filed with Alnylam's most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam's views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.
The scientific information referenced in this news release relating to givosiran is preliminary and investigative. Givosiran has not been approved by the U.S. Food and Drug AdministrationEuropean Medicines Agency, or any other regulatory authority and no conclusions can or should be drawn regarding its safety or effectiveness.


The Porphyria story of Victor LaFae with HCP

Porphyria story - HCP - Victor LaFae I’m told that I was a typical happy baby for the first few months of my life. I reached all my mile...