Rare Disease Report

Neurology

Data from Type 1 spinal muscular atrophy (SMA) treatment suggests efficacy and tolerability.  
CLN2 disease treatment reduces rate of clinical decline in children.  
 
IONIS-HTT data shows link between reduction in Huntingtin protein and clinical improvement. 
Nusinersen treatment proven to be efficient in spinal muscular atrophy (SMA). 
AbobotulinumtoxinA proves effective across multiple therapeutic uses.  
 
New data proves efficacy of relapsing multiple sclerosis (RMS) treatment.
 
Dr Ali Fatemi, MD, of Kennedy Krieger Institute and John Hopkins University, describes the “Awesome Disease” (LBSL) and how a young girl now inspires his research. 
 
The first patient was enrolled in a Phase 3 clinical trial of amifampridine phosphate (Firdapse) in patients with mmuscle-specific kinase (MuSK) antibody positive myasthenia gravis (MuSK-MG).
 
Positive Phase 1b/2a trial results for RT001 in patients with Friedreich’s ataxia were published this morning in the online journal Movement Disorders.
FDA exercises enforcement discretion regarding its ExeGen ATM MiniSwine model, clearing the genetically engineered (GE) to model ataxia telangiectasia (AT) for commercial use as a research tool.
 
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