Marisa Wexler, MS, senior science writer —

Catalyst Pharmaceuticals has secured a second U.S. patent covering Firdapse (amifampridine), an approved oral treatment for Lambert-Eaton myasthenic syndrome (LEMS). “We are pleased that this second patent for Firdapse has been issued and believe that it further enhances our intellectual property protection for Firdapse,” Patrick J. McEnany, chairman…

Catalyst Pharmaceuticals has teamed up with DyDo Pharma to develop and commercialize Firdapse (amifampridine) in Japan for the treatment of Lambert-Eaton myasthenic syndrome (LEMS). Under the terms of their agreement, DyDo will be responsible for funding all clinical, regulatory, marketing, and commercialization activities in Japan. In turn,…

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…

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…

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…

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.

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 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…

A $750,000 grant from the National Science Foundation will fund a project to build a better computer model of the communication between nerves and muscle cells. The project may have applications for Lambert-Eaton myasthenic syndrome (LEMS), a condition in which the immune system disrupts nerve-muscle communication. Nerve…