MDA is proud to have the support of Pfizer, a company working on several potential therapies for rare and genetic diseases such as spinal muscular atrophy (SMA), which is one of more than 40 neuromuscular diseases covered by MDA. Earlier this year, Pfizer announced that it had entered into a license agreement with the biotechnology firm Repligen to advance Repligen’s SMA program, including the investigational therapy RG3039, which was licensed in 2009 by Repligen from Families of SMA (FSMA). FSMA fully funded and directed the preclinical development work with an investment of more than $13 million. It was the very first industrial drug program ever done for SMA. MDA has helped to support the development of RG3039 through its translational research program.
“We are looking forward to participating in the MDA Scientific Conference,” said Michael Tones, Ph.D., of Pfizer’s Rare Disease Research Unit. “Inherited neuromuscular diseases such as spinal muscular atrophy and Duchenne muscular dystrophy are a key part of Pfizer’s rare disease research efforts. This is an exciting time in neuromuscular science, and MDA fulfills a unique role by supporting innovative science in this area.”
About 500 scientists, many of whom are at the vanguard of neuromuscular disease research, will attend the conference and present their newest findings about Duchenne muscular dystrophy, spinal muscular atrophy, ALS and additional neuromuscular diseases. Fueled by rapidly evolving biotechnology practices, an unprecedented number of new potential disease therapies have been developed.
“Five years ago, this meeting couldn’t have happened," said MDA Vice President of Research Jane Larkindale. "Advances in biotech research are moving quickly, and we now have promising new paths for treatment development. Our conference highlights projects from early-stage therapeutic targets through to clinical trial results. MDA appreciates the support Pfizer is providing for this conference and for disease research. We have been funding muscle disease research for more than 60 years, and the interest that Pfizer is showing in neuromuscular diseases, particularly demonstrated by the licensing of RG3039, is encouraging to us and to MDA families.”
In addition to the academic scientists who will attend the meeting, MDA is bringing together professionals from government, industry and the nonprofit sector to join the discussion about therapy development for neuromuscular disorders.
Conference co-chairs are: C. Frank Bennett, senior vice president for research at Isis Pharmaceuticals; and Eric Hoffman, director of the Research Center for Genetic Medicine at Children's National Medical Center in Washington, D.C.
MDA is the nonprofit health agency dedicated to finding treatments and cures for muscular dystrophy, ALS and related diseases by funding worldwide research. The Association also provides comprehensive health care and support services, advocacy and education.
In addition to funding more than 250 research projects worldwide, MDA maintains a national network of 200 medical clinics; facilitates hundreds of support groups for families affected by neuromuscular diseases; and provides local summer camp opportunities for thousands of youngsters living with progressive muscle diseases.