With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough ... Read more
Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s ... Read more
A rare case of non-small cell lung cancer (NSCLC) was discovered in a patient with paraneoplastic cerebellar degeneration and Lambert-Eaton myasthenic syndrome (PCD-LEMS) — the first report to link the cancer ... Read more
Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. ... Read more
Treatment with Firdapse (Amifampridine phosphate) or Ruzurgi (3,4‐diaminopyridine, or 3,4‐DAP) is effective and well-tolerated among people with Lambert-Eaton myasthenic syndrome (LEMS), even in the presence of immunosuppressants or other ... Read more
Diagnosing Lambert-Eaton myasthenic syndrome (LEMS) as early as possible is key for effectively managing the disease, according to a review study, which also highlights the need to test for ... Read more
A study provided an overview of the use of amifampridine (brand names Firdapse and the recently approved Ruzurgi), along with other treatment choices for Lambert-Eaton myasthenic syndrome, including those for resistant muscle ... Read more
A 71-year-old man has been diagnosed with both Lambert-Eaton myasthenic syndrome (LEMS) and thymic small cell cancer in a rare case study linking the two conditions. The study, titled ... Read more
Amifampridine-based medications are the most effective and safest options for managing symptoms of Lambert-Eaton myasthenic syndrome (LEMS), and should be chosen as the first-line management therapy for the rare ... Read more
Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board ... Read more
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