News

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,…

Using stimulated single-fiber electromyography is associated with an early and accurate diagnosis of Lambert-Eaton myasthenic syndrome, according to a case report. The study, “Stimulated single-fiber electromyography (sSFEMG) in Lambert-Eaton syndrome,” was published in the journal Clinical Neurophysiology Practice. Lambert-Eaton myasthenic syndrome (LEMS) is an autoimmune disease that…

Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced to seven years in federal prison for securities and wire fraud while heading San Diego-based Retrophin. As founder and CEO of another company (then known as Turing Pharmaceuticals), in late 2015 Shkreli bought the rights…

More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products Breakthrough Summit. The Oct. 15-16 event, sponsored by the National Organization for Rare Diseases (NORD), takes place at the…