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 as high as 350 million — they are thought to impact. Across countries, patients, caregivers and advocates will paint faces, wear…
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 myasthenic crisis,” was published in the journal Muscle & Nerve. LEMS and MG are…
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 pay out-of-pocket medical expenses. The Lambert-Eaton Myasthenic Syndrome Financial Assistance Program has more information on how to enroll at…
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— how disorders such as spinal muscle atrophy(SMA) run their course if untreated — the choice of “efficacy endpoints” in clinical trials, and how…
Lambert-Eaton myasthenic syndrome (LEMS) can develop as an immune-related adverse event caused by treatment with the anti-cancer therapy Opdivo (nivolumab), according to a case report. The study describes the case of a 73-year-old Japanese woman who started to…
Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of new but expensive therapies to treat rare diseases has never been greater. That’s the paradox faced by pharmaceutical companies as well as patient advocacy groups in…
The U.S. Food and Drug Administration has approved Firdapse (amifampridine phosphate), developed by Catalyst Pharmaceuticals, for adults with Lambert-Eaton myasthenic syndrome (LEMS), making it the first approved therapy in the U.S. for the condition. “There has been a long-standing need for a treatment for this rare…
Antibodies that recognize a protein called SOX2 that are specific markers of small cell lung cancer (SCLC) could be used to identify patients with Lambert-Eaton myasthenic syndrome (LEMS) who have this type of cancer, a study reports. The study, “The utility of anti-SOX2 antibodies for cancer…
Use of the immunotherapy alemtuzumab can promote the development of Lambert-Eaton myasthenic syndrome (LEMS), a rare case study in a patient with relapsing-remitting multiple sclerosis suggests. The study, “Lambert-Eaton myasthenic syndrome associated with alemtuzumab administration,” was published in the journal Multiple Sclerosis and Related Disorders.
With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Credit and affordable health insurance to future funding for rare disease research. Yet “whether Democrats take over the House or Senate,…
Get regular updates to your inbox.