Skip to main content

Highlights from the U.S. Conference on Rare Diseases and Orphan Products




Highlights from the U.S. Conference on Rare Diseases and Orphan Products

Role of Academic Centers, Looming Investment Crisis and More.

Jonathan Leff, MBA, Managing Director, Warburg Pincus, spoke in a special session on "Facing the Crisis in Biomedical Innovation: A Venture Investor's Perspective" on Day 2 of the conference.

Duke University Professor of Pediatrics Priya Kishnani, MD, told a standing-room-only audience the intensely moving story of the development of a treatment for infants and children with Pompe disease.  She focused on collaboration among academic researchers, industry and patient advocacy groups, describing the roles of Duke and Genzyme in developing an enzyme replacement therapy.

Lisa Coles, PhD, of the University of Minnesota's Center for Orphan Drug Research, described the expanding role of academic centers in drug discovery and development for rare disease R & D.  She presented strategies related to discovery of new chemical entities and drug repurposing.

FDA representatives and patient advocates discussed the changing role of patients at FDA and the agency's increased emphasis on interaction with patients.

On the Research/Regulatory track, sessions focused on endpoint development and post-marketing period opportunities for continue learning, among other things.  

The APF is active in the Rare Disease Community.


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

Comments

Popular posts from this blog

WHAT IS NEUROPATHIC PAIN?

What could it be?
Nerve Pain WHAT IS NEUROPATHIC PAIN? Neuropathic or nerve pain (NP) is a long-term or chronic pain disease that results from nerve damage. It can be caused by different diseases or conditions. Worldwide nerve pain affects as many as 26 million people. Neuropathic or nerve pain may affect larger areas of the body or it can be restricted to a smaller area, in this case it is called localized neuropathic pain (LNP). WHAT CAUSES NEUROPATHIC PAIN? Neuropathic or nerve pain may occur in the absence of an obvious visible cause (e.g. an accident, an injury, a chemical burn). There are several external situations that can directly damage nerves and lead to neuropathic pain, such as: Amputation of a limb (phantom pain, stump pain)Surgery (scar pain, post-surgery pain)Trauma or accident Neuropathic pain is also a common complication of other diseases, including nerve damage after shingles or herpes zoster infection (postherpetic neuralgia or PHN), nerve damage after HIV infectio…

GLOBAL PORPHYRIA ALLIANCE

GLOBAL PORPHYRIA ALLIANCE The APF is proud to support our members that live across the globe. Many countries around the world have developed organizations to support their local communities. We hope that enjoy the same opportunity to communicate with one another, develop friendships, and learn about porphyria. Here are links to patient advocacy groups worldwide that offer support to individuals impacted by porphyria. Argentina: Centro de investigaciones sobre porfirinas y porfirias | More informationAustralia: Porphyria Association, Inc. | More informationBrazil: Associação Brasileira de Porfiria | More informationCanada: Canadian Association for Porphyria | More informationColombia: Fundación Colombiana Para La Porfiria | More informationDenmark: Porfyriforeningen.dk (Porphyria Association Denmark)Denmark: Porfyrier.dk (Danish Porphyria Support Group)England: British Porphyria AssociationFinland: Finnish Porphyria Support Group France: Association Française des Malades Atteints de Por…

Happy Winter Season!

                  Happy Winter Season!