Rare Disease Report


This morning, it was announced that Invitae Corporation and Sarepta Therapeutics will be expanding its partnership to continue assisting clinicians in identifying patients with Duchenne muscular dystrophy (DMD).
Cytokinetics, Incorporated and The ALS Association continue their partnership in an effort to fight ALS.
This morning, Acceleron Pharma announced that the US FDA has granted Fast Track designation to ACE-083 for the treatment of patients with facioscapulohumeral muscular dystrophy (FSHD).
This morning, Capricor Therapeutics, Inc. announced that the HOPE-2 clinical trial has been initiated at UC Davis Medical Center in Sacramento, CA.
Data from Type 1 spinal muscular atrophy (SMA) treatment suggests efficacy and tolerability.  
At the 70th AAN Annual Meeting this morning Summit Therapeutics plc presented new 24-week interim data from PhaseOut DMD, its Phase 2 trial of ezutromid in Duchenne.
The first patient was enrolled in a Phase 3 clinical trial of amifampridine phosphate (Firdapse) in patients with mmuscle-specific kinase (MuSK) antibody positive myasthenia gravis (MuSK-MG).
According to data presented at the Annual Neuromuscular Translational Research Conference, researchers found that repeat dosing of CAP-1002 results in increased exercise performance in a disease model of Duchenne muscular dystrophy.
Pfizer announced that it has begun a Phase 1b clinical trial for PF-06939926, its mini-dystrophin gene therapy candidate, intended for boys with Duchenne muscular dystrophy.
Results from a study published in Nature Communications offer potential treatment targets for melorheostosis, often referred to as “dripping candle wax” bone disease.
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