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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, “…

As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking.  But exactly how expensive — in terms of direct and indirect costs across rare disease populations — might still come as a surprise: almost…

Editor’s note: This story was updated March 31, 2021, to clarify that Firdapse is currently the only therapy approved in the U.S. to treat adults with LEMS. Ruzurgi also is approved in the U.S. to treat children and adolescents, ages 6–16, with LEMS. Development of a new long-acting formulation of Firdapse…

In the U.S., treatments for rare diseases such as Lambert-Eaton myasthenic syndrome (LEMS) account for only 11% of total medical invoice spending in the country, according to a recent report. The report, “Orphan Drugs in the United States: Rare Disease Innovation and Cost Trends Through 2019,” is…

Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, blue, and purple lights side-by-side? These are the colors most often used to represent Rare Disease Day. This…

For the more than 25 million Americans living with rare diseases, including Lambert-Eaton myasthenic syndrome (LEMS), medical emergencies can pose unique challenges: because these disorders are by definition rare, first responders or emergency personnel may not be aware of a person’s condition, or familiar with the appropriate treatment.