News

Europe Awaits Proposed New Framework for Sharing Health Data

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…

Generic Amifampridine SERB Wins EMA Endorsement To Treat LEMS

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…

LEMS Case Shows Need for Testing To Prevent Treatment Delays

The case of an 8-year-old girl with Lambert-Eaton myasthenic syndrome (LEMS) who had no signs of a tumor, but had progressive muscle weakness, shows the need for comprehensive analysis and thorough investigation to prevent treatment delays. The report, “Lambert-Eaton myasthenic syndrome in a young girl,” was published in…

Increased Education Crucial to Improving Rare Disease Care, Survey Finds

Healthcare providers involved in diagnosing and treating rare diseases believe that increased physician education and collaboration with specialized facilities will have the greatest positive impact on treating these conditions over the next five years, according to results from a 2021 survey. Definitive Healthcare, a healthcare commercial intelligence company, conducted…

#RAREis Representation Program Promotes Equity, Diversity

Horizon Therapeutics has launched its #RAREis Representation program aimed at increasing diversity, equity, and inclusion among patients with rare diseases. There are about 400 million people worldwide living with a rare disease; for many of them, access to diagnosis, care, and treatments can be challenging. Accessing better care depends on…