Rare Disease Report


More treatments are needed for ALS, beyond the available riluzole and edaravone.
Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.
The phase 3 trial evaluating ZX008 as a treatment for Dravet syndrome met its primary endpoint.
The FDA has granted orphan drug designation to Acceleron Pharma Inc for its product, ACE-083, for the treatment of facioscapulohumeral muscular dystrophy (FSHD).
The FDA’s approval of Brineura in April 2017 was a landmark moment for patients with the CLN2 form of the disease, but those with the other 13 forms of Batten are still waiting.
Friedreich’s ataxia treatment, omaveloxolone, received orphan drug designation from the European Commission.  
An isotopic-reinforced polyunsaturated fatty acids shows a reduction in cognitive deficits in a preclinical in vivo model of Huntington disease.
Several promising new treatment developments are offering a ray of hope to Huntington’s patients, their at-risk children, and the clinicians who treat them.
Similar to the phase 2 MoveDMD trial, the phase 3 POLARIS trial will share the same patient population and functional endpoints.
Skyhawk Therapeutics and Celgene Corporation have entered into a 5-year partnership to discover, develop, and commercialize small molecule treatment options for patients with neurological diseases.
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