News

A total of 30 patient advocacy groups working to further understanding and the needs of people living with rare diseases have been selected to receive a Horizon Therapeutics‘ #RAREis Global Advocate Grant. Winners of this year’s inaugural awards are spread across nine countries and represent a total of 29 rare…

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…

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,…