News

The Muscular Dystrophy Association (MDA) is encouraging the U.S. Centers for Disease Control and Prevention (CDC) to recommend that people living with neuromuscular diseases (NMDs) have early access to any federally approved COVID-19 vaccine. The MDA made its request in a letter to members of the…

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) has launched an initiative across the U.S. Its goal is to establish a Rare Disease Advisory Council (RDAC)…

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about how their respective organizations came to be, as well as the benefits of creating patient registries and how they can help…

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases. “The RareLaunch program is central to NORD’s mission and history — community…

A recent case study of a woman with Lambert-Eaton myasthenic syndrome (LEMS) linked to lung adenocarcinoma — a type of cancer of the mucus-producing gland cells in the lungs — underscores the importance of cancer screening when LEMS is suspected. According to the researchers, only five cases of…

A photo of a bespectacled young boy, his red baseball cap slightly askew as he  enjoys time outside, will be featured on the front cover of an upcoming calendar in the “Same But Different” contest to raise awareness about rare disorders. “A Lovely Day Out in Kew Gardens,”…

Firdapse (amifampridine) is now widely available in Canada as a treatment for adults with Lambert-Eaton myasthenic syndrome (LEMS), according to KYE Pharmaceuticals, which markets the oral therapy. Health Canada, the country’s medicines regulatory agency, approved Firdapse, sold as a 10 mg tablet, earlier…

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…

The U.S. Patent and Trademark Office has issued a new patent to Catalyst Pharmaceuticals covering the company’s rights to Firdapse (amifampridine), a treatment for Lambert-Eaton myasthenic syndrome (LEMS). The patent (No. 10,793,893), “Methods of administering 3,4-diaminopyridine,” expires on April 7, 2034. Catalyst’s rights over Firdapse…