News

In a business update amid the COVID-19 pandemic, Catalyst Pharmaceuticals said it has ample supplies of Firdapse (amifampridine), its treatment for Lambert-Eaton myasthenic syndrome (LEMS). Besides having no known disruptions to Firdapse production, the company assured its treatment inventories will last at least through June 2021. Catalyst’s U.S.

A group of experts have provided a series of recommendations for people with myasthenia gravis (MG) and Lambert-Eaton myasthenic syndrome (LEMS) who rely on immunosuppressants to help manage their condition during the COVID-19 outbreak. Their guidelines, titled “Guidance for the management of myasthenia…

Catalyst Pharmaceuticals says the COVID-19 pandemic is not likely to affect North American supplies of Firdapse (amifampridine), its Lambert-Eaton myasthenic syndrome (LEMS) treatment. “We assure the LEMS community that we have product available and anticipate no disruptions to our manufacturing facilities, as they are all…

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. Now, the good news: You already know how to face loneliness and adversity — qualities that make you far stronger…

The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. The event’s sponsor, the National Organization for Rare Disorders (NORD),…

Emotional resilience is a term used to describe how people respond to changes in life and their ability to adapt to stressful situations. However, a person being more resilient than another doesn’t mean they don’t experience or feel the stress. Rather, it refers to how quickly they can adapt…

Timely cancer treatment helped stabilize a 63-year-old man for more than six years who had lung adenocarcinoma associated with Lambert‐Eaton myasthenic syndrome, unlike the poor prognosis seen in prior cases, a study reports. The study, “Successful treatment of advanced lung adenocarcinoma complicated with Lambert‐Eaton myasthenic syndrome:…

Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see the approval of 1,000 new rare disease therapies by 2027. That group, the International Rare Diseases Research Consortium (IRDiRC) —…

Catalyst Pharmaceuticals expects to report 2019 net product revenues of $102 million for Firdapse (amifampridine), the first and only therapy available for treating the symptoms of Lambert-Eaton myasthenic syndrome (LEMS). In addition, the biopharmaceutical company anticipates 2020 Firdapse net product revenues of $135 million to $155…

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…