Forest Ray PhD,  —

Global data standards for conducting research on rare diseases, such as Lambert-Eaton myasthenic syndrome (LEMS), are being developed by a new partnership between the Clinical Data Interchange Standards Consortium (CDISC) and the National Organization for Rare Disorders (NORD). Rare disorders in the U.S. are defined as those affecting fewer than…

The Orphan Drug Act (ODA) has spurred the development of treatments for rare diseases, such as Lambert-Eaton myasthenic syndrome (LEMS), according to a recent report. Yet, patient advocates say that a better balance between development incentives and competition might benefit the rare disease community. The report, “…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

Treatment with immunoglobulins, or antibodies — proteins that help fight off infections — successfully reduced symptoms of Lambert-Eaton myasthenic syndrome (LEMS) in a woman receiving immunotherapy for a rare type of skin cancer. Her case suggests that immunoglobulin therapy may prove an effective option for treating patients with…

Symptoms of Fisher syndrome can lead doctors to also diagnose asymptomatic Lambert‐Eaton myasthenic syndrome (LEMS), according to a case study in Japan. The report, “A case presenting electrophysiological and immunological characteristics of Fisher syndrome and Lambert‐Eaton myasthenic syndrome,” was published in the journal Muscle & Nerve.

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…

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…

Health Canada has approved the use of oral Firdapse (amifampridine) to treat people with Lambert-Eaton myasthenic syndrome (LEMS). Catalyst Pharmaceuticals‘ request for Firdapse’s approval in Canada was assigned priority review. The oral therapy (given in 10 mg tablets) is already available for…

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in the United States. People with rare diseases know that the impacts of such conditions extend beyond just medical…