Rare Disease Report

Neurology

Biohaven Pharmaceutical has established an expanded access program (EAP) with sublingual BHV-0223 for patients with amyotrophic lateral sclerosis (ALS).
DNAtrix will report data from an ongoing Phase 1 trial of DNX-2401 in pediatric patients with newly diagnosed diffuse intrinsic pontine gliomas (DIPG), which indicated efficacy through a prolonged survival rate. 
Alnylam Pharmaceuticals has achieved delivery of novel small interfering RNA (siRNA) conjugates to the central nervous system and intends to advance a pipeline of investigational RNAi therapeutics into clinical development.
A human clinical trial grant totaling $750,000 was awarded this morning by the Muscular Dystrophy Association (MDA) to Massachusetts General Hospital, with the hope that it will accelerate biomarker research for amyotrophic lateral sclerosis (ALS).
Bruce Wang, MD, a porphyrias specialist and assistant professor in the gastroenterology division in the department of medicine at University of California San Francisco (UCSF) and UCSF Porphyria Center and the Porphyrias Consortium, explains acute hepatic porphyrias (AHPs).  
According to new researche, a widely-used cholesterol-lowering drug increased a precursor of high-density lipoprotein (HDL) in models that mimicked liver cells of patients with Friedrich’s ataxia (FA).
Orchard Therapeutics announced that its gene therapy candidate, OTL-200, has been granted Rare Pediatric Disease designation for the treatment of metachromatic leukodystrophy.
A Rare Pediatric Disease designation was granted by the U.S. FDA this morning to Cellectar Biosciences for CLR 131, intended for the treatment of neuroblastoma.
As stated in article published in the clinical journal Neurobiology of Aging, a team of researchers has discovered that elevated levels of certain types of lipids in the brain could be an early presentation of Parkinson’s disease.
This morning, top-line data from a confirmatory Phase 2 study of NP001 in ALS were reported, and showed that the trial did not meet its primary or secondary endpoints.
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