Mary Chapman, features writer —

While unhappy with shortcomings in the legislation, the National Organization for Rare Disorders (NORD) nonetheless is applauding a year-end U.S. spending package that included several programs and reforms crucial to those with rare diseases, including Lambert-Eaton myasthenic syndrome (LEMS). As part of an expansive $1.7 trillion package, signed…

The National Organization for Rare Disorders (NORD) is requesting applications from patient advocacy organizations to host new or existing patient registries for rare disorders, such as Lambert-Eaton myasthenic syndrome, on its IAMRARE platform. To expand its collection of patient-reported data, which could lead to new treatments and…

The National Organization for Rare Disorders (NORD) is leading an effort by organizations representing or treating people with rare diseases, all calling on federal lawmakers to include key U.S. Food and Drug Administration (FDA) programs and reforms in a year-end spending bill. A letter written by NORD and signed by 126…

Catalyst Pharmaceuticals has been named BioFlorida’s David J. Gury Company of the Year for its breakthrough Lambert-Eaton myasthenia syndrome (LEMS) treatment Firdapse (amifampridine). BioFlorida, which represents Florida’s life sciences industry, recognizes a single company each year for significant milestones and achievements that advance life sciences in the…

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…

A federal appeals court in the U.S. has issued a mandate that effectively vacates the 2019 regulatory approval of Jacobus Pharmaceutical’s Ruzurgi for the treatment of children with Lambert-Eaton myasthenic syndrome (LEMS). The ruling, by the U.S. Court of Appeals for the 11th Circuit, directs a lower district court…

At its first virtual investor event, biotech company Centogene set a bold mission: to cure 100 rare diseases within the next decade. A leader in the field of genetic diagnostics, Centogene used the June 22 event to present its strategic priorities, outlining its plans to speed the discovery…

To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health information system, the EveryLife Foundation for Rare Diseases is presenting a first-of-its-kind resource guide. The foundation created the…

Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the National Organization for Rare Disorders (NORD) and set this year for June 26-27. The conference brings together patients,…

Equine-assisted therapy involves activities with horses to promote the physical and mental health of riders. For people with Lambert-Eaton myasthenic syndrome (LEMS), this kind of therapy may help to improve balance, flexibility, and other functions, and has also been shown to provide an emotional boost. What is LEMS? LEMS…