Is New Type 1 SMA Treatment in Sight?
Data from Type 1 spinal muscular atrophy (SMA) treatment suggests efficacy and tolerability.
CLN2 Disease Treatment Reduces Rate of Clinical Decline in Children
CLN2 disease treatment reduces rate of clinical decline in children.
IONIS-HTT Data Shows Link Between Reduction in Huntingtin Protein and Clinical Improvement
IONIS-HTT data shows link between reduction in Huntingtin protein and clinical improvement.
Nusinersen Proven to be Effective in SMA
Nusinersen treatment proven to be efficient in spinal muscular atrophy (SMA).
AbobotulinumtoxinA Proves Effective Across Multiple Therapeutic Uses
AbobotulinumtoxinA proves effective across multiple therapeutic uses.
New Data Proves Efficacy of Relapsing Multiple Sclerosis Treatment
New data proves efficacy of relapsing multiple sclerosis (RMS) treatment.
Physician Perspective on LBSL
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.
First Patient Enrolled in Phase 3 Trial of Firdapse in MuSK Antibody Positive Myasthenia Gravis
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 Results from Friedreich's Ataxia Trial Published
Positive Phase 1b/2a trial results for RT001 in patients with Friedreich’s ataxia were published this morning in the online journal
FDA Clears Rare Neurological Disease Model for Commercial Research Use
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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