News

A Russian military plane crash near Tetiana Zamorska’s home in Kyiv, Ukraine, was a sign that it was time for her and her family to leave. The treacherous, 34-hour pilgrimage that ultimately brought the group of eight by car to temporary accommodations in neighboring Poland last month was physically and emotionally difficult,…

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…

The National Organization for Rare Disorders (NORD) has updated its State Report Card to make it more digitally friendly and added telehealth to its categories of rare disease policy issues in a nod to its increased use during the ongoing COVID-19 pandemic. NORD’s report card project began seven…

A branch of the European Medicines Agency (EMA) has recommended that Amifampridine SERB, a generic version of Firdapse, be approved to treat adults with Lambert‑Eaton myasthenic syndrome (LEMS) in the European Union. The recommendation, from the EMA’s Committee for Medicinal Products for Human Use (CHMP), will now…

The case of an 8-year-old girl with Lambert-Eaton myasthenic syndrome (LEMS) who had no signs of a tumor, but had progressive muscle weakness, shows the need for comprehensive analysis and thorough investigation to prevent treatment delays. The report, “Lambert-Eaton myasthenic syndrome in a young girl,” was published in…

A simple, noninvasive test on the skin, called skin sympathetic response, can be used to confirm impairments in the autonomic nervous system — the non-conscious part of the nervous system that controls several bodily functions — in people with Lambert-Eaton myasthenic syndrome (LEMS), according to a case study. The…

Patient registries are a hot topic of rare disease research and many organizations are taking advantage of this resource by signing up their patient communities and connecting with researchers. Eric Sid, MD, program officer for the Office of Rare Diseases Research (ORDR), said it is difficult to estimate how…

The Federal Court of Canada has issued a decision that, for the second time, sets aside Health Canada’s approval of Ruzurgi (amifampridine) to treat Lambert-Eaton myasthenic syndrome (LEMS) in patients 6 and older. The decision is a legal victory for Catalyst Pharmaceuticals and its Canadian sublicensee, KYE…

Horizon Therapeutics has launched its #RAREis Representation program aimed at increasing diversity, equity, and inclusion among patients with rare diseases. There are about 400 million people worldwide living with a rare disease; for many of them, access to diagnosis, care, and treatments can be challenging. Accessing better care depends on…