Rare Disease Report

Neurology

Aquestive Therapeutics’ clobazam (SYMPAZAN) oral film has been approved by the US Food and Drug administration (FDA) for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) in patients aged 2 years or older.
 
Here’s a round-up of recent designations granted by the FDA to products developed to treat rare diseases.
Investigators in the United Kingdom have found that a simple blood test reveals the earliest signs of Huntington’s disease, and may help scientists working on developing new therapeutics for the genetic disease.
New study serves as a reminder of how little is known about the clinical and biological manifestations of this form of Huntington's disease.
 
Positive interim clinical data from Genentech’s 2-part pivotal FIREFISH and SUNFISH trials evaluating risdiplam (RG7916), a potential spinal muscular atrophy (SMA) treatment, have been released.
 
Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.
In a recent interview with Rare Disease Report®, Hartmann Wellhoefer, MD, discussed the biggest challenges faced and advances being made in the fight against MPS.
 
The FDA has granted an orphan drug designation to Q Therapeutics Inc.’s Q-Cell for the treatment of transverse myelitis.
Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.
Fingolimod has been linked with a lower rate of relapse and less accumulation of lesions on MRI over a 2-year period compared with interferon beta-1a in a phase 3 PARADIGMS trial. 
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