As the search continues for SMA treatments and a cure, MDA is working to help those living with the disease today
MDA is working to improve life span and quality of life for people with spinal muscular atrophy (SMA). Those efforts include the creation of the MDA clinical neuromuscular disease registry, promotion of newborn screening for SMA and publication of a family friendly version of SMA standard of care guidelines.
MDA clinical neuromuscular disease registry
People with FA, or other disorders classified as ataxias, are encouraged to enroll in the CoRDS ataxia patient registry, which aims to advance ataxia research
The Coordination of Rare Diseases at Sanford (CoRDS) national rare disease registry is now hosting an ataxia patient registry for people with Friedreich's ataxia (FA) or other disorders classified as ataxias (conditions that cause problems with balance or coordination).
Disease registries are seeking participants with CMD, FSHD, LGMD, MMD and Pompe disease
FSHD, MMD: Registry remains open
The University of Rochester Medical Center in New York state would like to remind people with facioscapulohumeral muscular dystrophy (FSHD) or myotonic muscular dystrophy (MMD, or DM), and their families, about its registry to advance research in these two disorders.
Featured in this update:
Families, experts meet at BMD ConferenceThree trials study blood-vessel-dilating drugs in BMD, DMDResults of daily, weekly prednisone treatment about the same in DMD