Rare Disease Report


NBC News chief foreign correspondent Richard Engel's most demanding role has taken place off-camera. He and his wife, Mary Forrest, support each other in raising their 2-year old son, Henry, a patient with Rett syndrome.
This morning, Aquestive Therapeutics, Inc. announced that the U.S. FDA has granted orphan drug designation to Riluzole Oral Soluble Film (riluzole OSF) for the treatment of ALS.
PTC Therapeutics has announced that Part 1 of the FIREFISH study has shown that RG7916, a Type 1 spinal muscular atrophy (SMA) drug, to be safe and well-tolerated in infants and babies with Type 1 SMA.
Alnylam announced that its Marketing Authorisation Application for patisiran was accepted by the European Medicines Agency.
New data concludes that neurofeedback training could potentially increase brain connectivity in patients with Huntington’s disease (HD).
The latest #ALSPepperChallenge video comes from Rare Disease Report. Participate today to raise awareness for ALS.
The peer-reviewed clinical journal Neurology has published the Phase 2 trial results for arimoclomol in patients with SOD1 amyotrophic lateral sclerosis (ALS).
On his personal website Monday, legendary musician Neil Diamond revealed that he has been diagnosed with Parkinson’s disease.
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.
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