Angie Cone talks about the nonprofit Chive Charities that is improving life for rare disease patients.
LPG1837 is a candidate drug for the treatment of the Class III mutation in cystic fibrosis.
Timothy Wood, PhD, of Greenwood Genetic Labs summarizes the tests commonly used to help clinicians diagnose patients with Morquio A, a rare lysosomal storage disease.
Jim O'Heir and Chris Hawkey were among the celebrities reading the play ‘Four Decades, One Community’ about the history of the TSC Community.
Bonner Paddock talks about the origins of his non-profit organization, OM Foundation, that is improving the lives of disabled school children in both in the United States and Africa.
Chris Garabedian, former president and CEO of Sarepta Therapeutics talks about the common practice of many advocacy groups being available for one rare condition.
MyoKardia, Inc., announced initial clinical data from two Phase 1 trials of MYK-461, which targets the underlying cause of hypertrophic cardiomyopathy (HCM).