Marisa Wexler, MS,  senior science writer—

The U.S. Congress has passed five-year extensions for several user fee programs of the U.S. Food and Drug Administration (FDA), as part of legislation that will keep the government funded through December. The move was welcomed by the National Organization for Rare Disorders (NORD), which stated these programs…

The U.S. Food and Drug Administration (FDA) has expanded its approval of Firdapse (amifampridine) to cover pediatric patients, its maker Catalyst Pharmaceuticals announced. This means the therapy is approved in the U.S. to treat people with Lambert-Eaton myasthenic syndrome (LEMS) ages 6 and older. “We are very pleased…

Repeated testing of electrical signaling in muscles before and after exercise is a key test for diagnosing Lambert-Eaton myasthenic syndrome (LEMS), a new case report highlights. The case was discussed at the 32nd International Congress of Clinical Neurophysiology (ICCN) of the IFCN, Sept. 4–8 in Geneva, Switzerland, in a…

The prevalence of Lambert-Eaton myasthenic syndrome (LEMS) in Japan is similar to that of other countries, affecting approximately 2.7 out of every one million people in the country. That is according to a study, “Nationwide survey of Lambert-Eaton myasthenic syndrome in Japan,” published in the journal…

The diagnostic journey of a man in his early 30s with progressive muscle weakness that was ultimately determined to be due to Lambert-Eaton myasthenic syndrome (LEMS) was described in a recent case report. The report, “Progressive motor quadriparesis with wasting: a diagnostic conundrum,” was published in BMJ Case…

In a deal that ends a three-year U.S. court battle over patent infringement claims for Lambert-Eaton myasthenic syndrome (LEMS) medications, Catalyst Pharmaceuticals will acquire the rights to the LEMS therapy Ruzurgi. Catalyst announced it has reached a settlement with Jacobus Pharmaceutical and PANTHERx Rare on its patent…

A company called Vibe Biotechnology has launched to build a community of patients, scientists, and other partners to find cures for rare diseases like Lambert-Eaton myasthenic syndrome (LEMS). “The challenge for rare diseases isn’t necessarily finding a treatment — it’s funding it,” Alok Tayi, PhD, co-founder and CEO of…

Long-term treatment with Firdapse is generally well-tolerated by people with Lambert-Eaton myasthenic syndrome (LEMS), according to a study of patient registry data. “The European LEMS registry aimed to collate observational safety data on treatments offered to patients with LEMS, particularly [Firdapse], which was approved in the year before…

The potential risks and benefits of several policy changes that could affect the landscape of developing treatments for rare diseases like Lambert-Eaton myasthenic syndrome (LEMS) were discussed in a recent white paper. The paper, “The Next Generation of Rare Disease Drug Policy: Ensuring Both Innovation and Affordability,” was…

The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…