News

NORD Applauds New US Legislation for Rare Disease Funding

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…

NORD Hopes to Add 4 Patient Registries to IAMRARE Platform

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…

Cancer Immunotherapy Tecentriq Tied to Higher LEMS Risk in Study

People with small cell lung cancer (SCLC) treated with the immune-based cancer therapy atezolizumab are at an increased risk of developing Lambert-Eaton myasthenic syndrome (LEMS), according to a database analysis of adverse events. The analysis was detailed in a study, “Increased risk of Lambert-Eaton myasthenic syndrome (LEMS)…

NORD, Others Urge Congress to Fully Fund FDA Rare Disease Programs

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’s Revenue From Firdapse Tops $50M in 3rd Quarter 2022

Catalyst Pharmaceuticals earned $57.2 million in the third quarter of 2022 from sales of Firdapse (amifampridine), its treatment for Lambert-Eaton myasthenia syndrome (LEMS). According to Catalyst, this represents an all-time high net revenue from Firdapse, a 59.3% year-over-year increase and a gain of 7.8% compared with the…

Firdapse’s Catalyst Pharmaceuticals Named BioFlorida’s Top Company

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…

LEMS Tied to Rare Skin Cancer Successfully Treated: Case Study

Lambert-Eaton myasthenic syndrome (LEMS) arose in association with a rare type of aggressive skin cancer called Merkel cell carcinoma (MCC), a case study reported for the first time. The patient was successfully treated with a combination of radiotherapy, chemotherapy, and immunotherapy, which may be a suitable strategy for those…