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March 26, 2019March 26, 2019

Mouse Model of LEMS Provides New Tool to Develop Therapies

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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 effective testing of treatment candidates. ... Read more

March 14, 2019March 14, 2019

Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn

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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 scheduled, at the end of ... Read more

March 12, 2019March 12, 2019

Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist

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With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases and the expensive therapies needed ... Read more

March 11, 2019March 11, 2019

NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research

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Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet only 5 percent of the estimated 7,000 rare diseases known ... Read more

March 4, 2019March 4, 2019

Firdapse Found to be Effective for Lambert-Eaton Myasthenic Syndrome, Phase 3 Trial Shows

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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 trial. The results were published ... Read more

February 25, 2019February 25, 2019

WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April

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The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have ... Read more

February 20, 2019

Vienna to Host RARE2019 Meeting on Rare Diseases

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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 Treatments in Rare Diseases. The ... Read more

February 12, 2019February 12, 2019

Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide

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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 of people — estimates run ... Read more

February 11, 2019February 11, 2019

Repetitive Nerve Stimulation Test Could Be Useful to Diagnose LEMS, Myasthenic Crisis, Study Shows

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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, “Repetitive nerve stimulation test in ... Read more

February 4, 2019February 4, 2019

LEMS Patients Can Get Financial Aid from The Assistance Fund

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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 program to help eligible patients ... Read more

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