News

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in  grants, totaling up…

Plasmapheresis, or plasma exchange, may be an effective way of managing small cell lung cancer-associated Lambert-Eaton myasthenic syndrome (LEMS) and paraneoplastic cerebellar degeneration (PCD), combined with peripheral nerve damage, a case report suggests. The simultaneous development of LEMS and PCD is usually associated with small cell lung cancer (SCLC)…

RARE-X, a collaborative platform for patient-controlled data collection, global data sharing and analysis, is partnering with the Broad Institute of MIT and Harvard to advance and support the discovery of new treatments for 9,500 rare diseases, including Lambert-Eaton myasthenic syndrome (LEMS). The collaboration will leverage Broad Institute’s…

The case of a man in whom a neuroendocrine tumor was found to be associated with Lambert-Eaton myasthenic syndrome (LEMS) underscores the importance of early diagnosis and treatment of the two rarely associated conditions. “To the best of our knowledge, this…

Lambert-Eaton myasthenic syndrome (LEMS) may be associated with a rare type of skin cancer called Merkel cell carcinoma (MCC), according to a case study which also highlighted the need for multidisciplinary care in such complex cases. The case report, “Lambert-Eaton Myasthenic Syndrome and Paraneoplastic Cerebellar Degeneration…

The Canadian Agency for Drugs and Technologies in Health (CADTH) has recommended that Ruzurgi (amifampridine) be reimbursed for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in individuals 6 and older, but only if certain conditions are met. These include patients for whom the medication has been prescribed by…

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…

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…

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, “…