Mouse Model of LEMS Provides New Tool to Develop Therapies
An animal model of Lambert-Eaton myasthenic syndrome (LEMS) has enabled better understanding of what drives the hallmark reduction in acetylcholine release as well as ... Read more
Updates
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Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn
Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as ... Read more -
Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist
With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases ... Read more -
NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research
Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet only 5 percent of the ... Read more -
Firdapse Found to be Effective for Lambert-Eaton Myasthenic Syndrome, Phase 3 Trial Shows
Firdapse led to clinically significant improvements, compared with a placebo, in adult patients with Lambert-Eaton myasthenic syndrome (LEMS), according to full results of a Phase 3 ... Read more
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WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April
The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government ... -
Vienna to Host RARE2019 Meeting on Rare Diseases
About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city ... -
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 ... -
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 ... -
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 ... -
FDA Revising ‘Draft Guidance’ on Developing Treatments for Rare Diseases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 ...
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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