Rare Disease Report


This morning, Sobi, announced that the FDA has issued a Study may proceed letter for the first study in humans, thereby accepting the investigational new drug application for sobi003, and the regulatory agency granted Fast Track status to the product candidate.
uniQure was granted Orphan Medicinal Product Designation from the European Medicines Agency for AMT-130, an investigational gene therapy for the treatment of Huntington’s disease.
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.
Copyright © RareDR 2013-2018 Rare Disease Communications. All Rights Reserved.