Rare Disease Report

Neurology

New evidence suggests that Rasmussen’s encephalitis is an autoimmune disease.
The FDA has granted Orphan Drug Designation to AMO-04 for the treatment of Rett syndrome.
The FDA has granted orphan drug designation to GPLSCD01 for the treatment of limbal stem cell deficiency.
Manmeet Ahluwalia, MD, Head of Operations in the Brain Tumor Center at Cleveland Clinic, explains what a team approach should look like when treating brain tumors and other rare cancers. 
 
Upon unearthing rheumatoid arthritis’ epigenome, researchers at University of California San Diego School of Medicine and the Icahn School of Medicine at Mount Sinai have also discovered an overlap between the causes of rheumatoid arthritis and Huntington’s disease.
By adjusting protein levels that impact an important biological pathway, researchers improve heart function in mice with HD, shedding light on the biology of the disease.
Supported by positive data from an ongoing Phase 2/3 study, bluebird bio’s Lenti-D has been granted Breakthrough Therapy designation by the US FDA for the treatment of patients with cerebral adrenoleukodystrophy.
Harmony Biosciences, LLC, announced that the FDA has granted Breakthrough Therapy and Fast Track designations to its product, pitolisant, for the treatment of excessive daytime sleepiness and cataplexy in patients with narcolepsy.
 
Late Friday afternoon, Novartis announced that the U.S. FDA approved fingolimod (Gilenya) to treat relapsing multiple sclerosis (MS) in children and adolescents age 10 years and older.
AMO Pharma Limited announces the commencement of patient recruitment for an interventional study of AMO-01, an investigational Ras-ERK pathway inhibitor for the treatment of Phelan-McDermid syndrome (PMS).
 
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