Patients & Caregivers
Will Tirasemtiv be Effective in ALS? A Conversation with Fady Malik of Cytokinetics
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).
Sarepta Investing in CRISPR Technology to Develop DMD Drugs
Duke University and Sarepta Therapeutics are joining forces
Orphan Drug for Proteus Syndrome Gets Rare Pediatric Designation
Proteus syndrome is characterized by bones, skin, and other tissues growing out of proportion to the rest of the body.
Enrollment Completed in Phase 3 of Firdapse in Lambert-Eaton Myasthenic Syndrome
The LMS-003 Phase 3 trial evaluating Firdapse (amifampridine phosphate) in patients with Lambert-Eaton Myasthenic Syndrome (LEMS) has completed enrollment.
Stranger Things Star Helps to Form CCD Non-Profit
Gaten Matarazzo, star of the Netflix original series
, is using his star to help patients with cleidocranial dysplasia (CCD).
FDA Rejects PTC Therapeutics' Application for Duchenne Drug
A complete response letter means the FDA is unable to approve the drug application in its current form.
Myasthenia Gravis Treatment Gets First FDA Approval Since 1950s
Alexion Pharmaceuticals, Inc. announced that the U.S. FDA has approved Soliris (eculizumab) as a new treatment for adult patients with generalized myasthenia gravis.
FDA Reviewing Burosumab for Rare Form of Rickets
X-linked hypophosphatemia is a heritable form of rickets. There are currently no approved therapies for this phosphate wasting condition
Sarepta Debuts Golodirsen Data at World Muscle Society Meeting
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.
Newly Released Data from Phase 1/2 Shows Promise for Amicus' Pompe Drug
New data shows Amicus' Pompe drug improve the 6-minute walk test distance and other measures of motor function.
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