Note: This story was updated July 13, 2022, to correct the name of Rare-X’s CEO Charlene Son Rigby. Nonprofits, scientists, governmental organizations, and the rare disease drug development industry have long cited 7,000 as the average number of rare diseases in the world.
Sessions advocating for increased access to treatment for rare diseases, and the need for greater equity, were some of the featured highlights of the NORD “Living Rare, Living Stronger Patient and Family Forum.” Other key takeaways from the forum — hosted by the National Organization for Rare Disorders, known…
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…
Legislation to create a Rare Disease Advisory Council (RDAC) in Colorado has passed the state’s Senate and House and was recently signed into law by Gov. Jared Polis, making “the Centennial State” the 24th U.S. state to establish such a council and the 10th since the beginning of 2021. RDACs…
A rare case of Lambert-Eaton myasthenic syndrome (LEMS) triggered by an immune-related cancer therapy was recently described in a case study. Because immunotherapy is relatively new, patients receiving these treatments should be monitored for “unexpected and unknown side effects,” the researchers noted. The case study, “Lambert-Eaton…
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…
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