Rare Disease Report

Neurology

Cure CMD (Congenital Muscular Dystrophy) is pleased to announce the recipient of a grant co-funded with Muscular Dystrophy UK: Dr. Giséle Bonne at the INSERM-Myology Institute in Paris.
Stay up-to-date on the latest rare disease news by checking out our top 5 articles of the week.
The FDA has denied accelerated approval for Pompe disease treatment, AT-GAA. However, plans for a pivotal study are still underway, so approval could be forthcoming.
 
There’s no cure for Huntington’s disease, but a new study by investigators in Sweden is exploring potential new approaches to treating the neurodegenerative disease.
 
Stay up-to-date on the latest rare disease news by checking out our top 5 articles of the week.
Daniel Ontaneda, MD, provides insight into the implications of discovering a new subtype of multiple sclerosis: myelocortical MS.
The first part of a recent phase 1/2a trial has met its primary objectives, demonstrating safety & tolerability of SYNB1618 in healthy volunteers & identifying a suitable dose to evaluate in patients with PKU. 
The FDA has approved the first and only easy-to-swallow thickened riluzole liquid for the treatment of amyotrophic lateral sclerosis.
Bruce Trapp, PhD, discusses his team’s recent discovery of a new subtype of multiple sclerosis: myelocortical MS.
Spinal muscular atrophy patients can see benefits even from late initiation of treatment with the drug nusinersen, according to a new study.
 
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