Author Archives: Forest Ray PhD

Global Data Standards Being Developed for Rare Disease Research

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…

Report Examines Pros, Cons of Orphan Drug Status

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, “…

Fisher Syndrome Can Co-occur With Asymptomatic LEMS

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 Approved in Canada to Treat People With LEMS

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…