News

The National Organization for Rare Disorders’ (NORD) “Living Rare, Living Stronger Patient and Family Forum” is back in person on June 26 for a day of learning and networking in Cleveland, Ohio. The event, which brings together the rare disease community, will take place at the InterContinental Cleveland Conference…

Lawmakers in Connecticut agreed to set up a permanent Rare Disease Advisory Council (RDAC), an advisory body working to better address the unique needs of people with rare diseases like Lambert-Eaton myasthenic syndrome (LEMS). An effort to establish these councils across the U.S. and to optimize existing ones,…

Leaders of the U.S. House Energy and Commerce Committee and two senators have taken action to clarify the original intent of the orphan drug act (ODA) that the U.S. Food and Drug Administration (FDA) applies to help speed therapy development for rare diseases. Sens. Bill Cassidy, R-Louisiana, and Tammy Baldwin, D-Wisconsin,…

Blood levels of long non-coding RNA LOC338963 and its potential target AP3B2 — a protein in nerve cells — are significantly increased in people with cancer-associated Lambert-Eaton myasthenic syndrome (LEMS) relative to healthy people, LEMS patients without cancer, and people with cancer, a study shows. The data also suggest…

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…

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…