News

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…

Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest.    Two entries in November’s “Disorder: The Rare Disease Film Festival” will focus on what siblings go through, according to the San Francisco festival’s co-founder,…

Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such issues during a recent talk at the New York Genome Center. Minssen directs the Center for Advanced Studies in…

3,4‐diaminopyridine — the active ingredient of the only FDA-approved treatment for adults with Lambert-Eaton myasthenic syndrome (LEMS) — effectively targets muscles in rats and has a long-lasting effect in the body, a study says. The findings of the study, “Pharmacokinetics and Tissue Distribution of 3,4‐Diaminopyridine in Rats,”…

Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one of just six such people who’ve made it to adulthood. Neena Nizar doesn’t have to imagine. The 41-year-old English professor at Metro Community College in Elkhorn,…