Rare Disease Report
Patients & Caregivers


In 2018, uniQure N.V. expects to advance the clinical development of AMT-130, its investigational gene therapy for the treatment of Huntington’s disease.
Results from a study report that behavioral interventions, like environmental enrichment, can reduce the functional deficit experienced by patients with Rett syndrome.
Data published in the Orphanet Journal of Rare Diseases suggests that endurance training could have a positive impact in Huntington’s disease patients, much like it has in anyone else.
Adamas Pharmaceuticals, Inc. announced the full commercial launch of Gocovri (amantadine) extended release capsules for the treatment of dyskinesia in patients with Parkinson’s disease receiving levodopa-based therapy, with or without concomitant dopaminergic medications.
Acceleron Pharma has announced that Part 1 of the Phase 2 clinical trial of ACE-083, a drug used to increase muscle volume in facioscapulohumeral dystrophy (FSHD), has exhibited positive results.
Stephen Hawking, the notable British physicist and author of A Brief History of Time, celebrates his 76th birthday today, making him one of the longest surviving patients with amyotrophic lateral sclerosis (ALS).
Last week, Pfizer announced a new partnership with Sangamo. As part of the agreement, the sides will team to develop a potential gene therapy to treat ALS, or Lou Gehrig’s disease.
Two months after Ionis submitted its NDA to the U.S. FDA, the company has announced that the regulatory agency has accepted its investigational drug inotersen for Priority Review.
Recent data have found that the same gene editing platform can disable the defective gene responsible for amyotrophic lateral sclerosis in mice. The therapy, which extended the lifespan of the mouse models by 25%, delayed the onset of the muscle wasting which characterizes the disease.
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