Rare Disease Report
Patients & Caregivers


Revance Therapeutics’ lead drug candidate DaxibotulinumtoxinA for Injection (RT002) has been granted orphan drug designation by the FDA to treat cervical dystonia.
The 2017 Congenital Muscular Dystrophy (CMD) Scientific and Family Conference made for the largest-ever gathering of CMD-affected individuals, family members, and experts.
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
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