Taking the First Scary Step After My LEMS Diagnosis
Being diagnosed with Lambert-Eaton myasthenic syndrome (LEMS) after living over a decade with progressive neuromuscular symptoms was a relief, at first. For so long, ... Read more
Updates
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Triple-Timed Up-and-Go Test Effective in Assessing LEMS Severity, Trial Analysis Shows
The Triple-Timed Up-and-Go (3TUG) test is a rapid, non-invasive, reliable, and reproducible measurement for assessing muscle weakness in people with Lambert-Eaton myasthenic syndrome (LEMS), ... Read more -
World’s First Alport Stamp Is Macedonian Mom’s Latest Win for Rare Disease Patients
It wasn’t until Gordana Loleska’s son David was 14 years old that doctors in their native North Macedonia diagnosed his kidney, vision, and hearing ... Read more -
As a LEMS Patient, I’m Concerned About the Ongoing IVIG Shortage
Patients across the United States receive regular phone calls about their intravenous immunoglobulin (IVIG) treatments. Typically, these calls are to confirm scheduled treatments. However, ... Read more
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‘LEMme Tell Ya’ About Me and My Extremely Rare Disease
“Dawn, what you have is extremely rare. It’s so rare that most doctors won’t ... -
NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala
A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy ... -
Eurordis Unveils Integrated-care Initiative for Rare Disease Patients
Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to ... -
Catalyst Expands Firdapse’s License Coverage to Include Japan, Sues FDA Over Ruzurgi’s Approval
Catalyst Pharmaceuticals has taken the first step to expand its commercial territory to cover ...
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FDA Accepts Catalyst’s NDA for Firdapse with Priority Review Status for LEMS
News
The U.S. Food and Drug Administration has accepted Catalyst Pharmaceuticals’ new drug application (NDA) for Firdapse (amifampridine phosphate) for Lambert-Eaton myasthenic syndrome (LEMS), with priority review status. Firdapse is a formulation of amifampridine phosphate in development for the treatment of LEMS as ... Read more