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 highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.
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 to late-onset PCD-LEMS, according to researchers. Their study, “Non-small cell lung cancer associated with late-onset Lambert–Eaton myasthenic syndrome…
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. Disorder: The Rare Disease Film Festival strives to eventually host a film about every one of the nearly 7,000 rare…
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 medications that alter the responsiveness of the immune system, a review has found. The review study,…
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 small cell lung cancer in these patients. The review, “Lambert-Eaton Myasthenic syndrome: early diagnosis is key,”…
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 weakness. The review study, “Recent Advances and Therapeutic Options in Lambert-Eaton Myasthenic Syndrome,” was published…
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 “Thymic small cell carcinoma associated with Lambert–Eaton myasthenic syndrome,” appeared in The Annals of Thoracic Surgery. Thymic…
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 autoimmune disorder, a review proposes. The study, “Amifampridine for the treatment of Lambert-Eaton myasthenic syndrome,” was published…
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 of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…
A case report describes a rare event of tongue muscle atrophy (shrinkage) in a 48-year-old man with Lambert-Eaton myasthenic syndrome who also was diagnosed with small-cell lung cancer (SCLC). Following radiotherapy for the tumor, the patient fully recovered control of the tongue. The study, “Reversible tongue atrophy in Lambert-Eaton…
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