Rare Disease Report
Patients & Caregivers


On December 8, Cytokinetics will present the results of its phase 3 clinical trial testing the safety and efficacy of tirasemtiv in patients with amyotrophic lateral sclerosis (ALS).
Duke University and Sarepta Therapeutics are joining forces
Proteus syndrome is characterized by bones, skin, and other tissues growing out of proportion to the rest of the body. 
The LMS-003 Phase 3 trial evaluating Firdapse (amifampridine phosphate) in patients with Lambert-Eaton Myasthenic Syndrome (LEMS) has completed enrollment.
Gaten Matarazzo, star of the Netflix original series Stranger Things, is using his star to help patients with cleidocranial dysplasia (CCD).
A complete response letter means the FDA is unable to approve the drug application in its current form.
Alexion Pharmaceuticals, Inc. announced that the U.S. FDA has approved Soliris (eculizumab) as a new treatment for adult patients with generalized myasthenia gravis.
X-linked hypophosphatemia is a heritable form of rickets. There are currently no approved therapies for this phosphate wasting condition
Sarepta released top-line results from its phase 1/2 muscle biopsy study of golodirsen in Duchenne last month, but provided more detail in a poster presentation.
New data shows Amicus' Pompe drug improve the 6-minute walk test distance and other measures of motor function.
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