Vienna to Host RARE2019 Meeting on Rare Diseases
About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced ... Read more
Updates
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Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide
Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions ... Read more -
Repetitive Nerve Stimulation Test Could Be Useful to Diagnose LEMS, Myasthenic Crisis, Study Shows
A repetitive nerve stimulation test can help diagnose and differentiate between Lambert-Eaton myasthenic syndrome (LEMS) and myasthenia gravis (MG) patients undergoing a myasthenic crisis, a study shows. The study, ... Read more -
LEMS Patients Can Get Financial Aid from The Assistance Fund
In recognition of the economic burden that comes with a diagnosis of Lambert-Eaton Myasthenic Syndrome (LEMS), the Assistance Fund has opened a financial assistance ... Read more -
FDA Revising ‘Draft Guidance’ on Developing Treatments for Rare Diseases
The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with new guidance on natural history— ... Read more
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Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish
Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures ... -
Firdapse Approved in U.S. for Adults with Lambert-Eaton Myasthenic Syndrome
The U.S. Food and Drug Administration has approved Firdapse (amifampridine phosphate), developed by Catalyst Pharmaceuticals, for ... -
Anti-SOX2 Antibodies May Be Useful Markers of Small Cell Lung Cancer in LEMS Patients, Study Says
Antibodies that recognize a protein called SOX2 that are specific markers of small cell lung ... -
Treatment with Lemtrada Caused LEMS in Relapsing-Remitting MS Patient, Case Report Shows
Use of the immunotherapy alemtuzumab can promote the development of Lambert-Eaton myasthenic syndrome (LEMS), ... -
#NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections Approach
With the U.S. midterm elections now less than two weeks away, patient advocacy groups ...
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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