1st LEMS Awareness Day dubbed ‘fantastic’ by founding nonprofit
Catalyst, developer of Firdapse for LEMS, supported inaugural March 30 event
The inaugural Lambert-Eaton myasthenic syndrome (LEMS) Awareness Day — which will be held each year on March 30 to call attention to the rare autoimmune disorder — was “fantastic,” according to the nonprofit association that created the event.
“We appreciate everything everyone did for this first LEMS Awareness Day, 2024,” the U.S.-based LEMS Family Association stated on its website. “We are moved by how much we saw it impacted our LEMS community.”
Among the supporters of the first annual Awareness Day — what it called a “landmark event” — was Catalyst Pharmaceuticals, which markets Firdapse (amifampridine), the sole oral treatment approved in the U.S. for reducing LEMS symptoms.
The company had announced its support in the days leading up to the occasion, with Richard J. Daly, Catalyst’s CEO, saying in a press release that the company was “proud to stand alongside the LEMS Family Association in honoring this pivotal event.”
Video marking 1st LEMS Awareness Day on nonprofit’s website
The new awareness campaign hopes to inform and educate the general public, as well as lawmakers, scientists, industry representatives, and health professionals. LEMS is thought to affect between 3,600 and 5,400 U.S. residents.
“In the realm of rare diseases, awareness is the key that unlocks paths to diagnosis and treatment,” Daly said.
“By shedding light on LEMS, we pave the way for better support, understanding, and care for those who need it most,” Daly added.
The nonprofit was founded in 2022 in Texas by LEMS patients to support researchers and those living with the disease. It also aims to increase awareness, and has a video of the inaugural event on its website.
“Creating an awareness day for LEMS is a huge win for our community,” said Price Wooldridge, the association’s founder and a board member. “This brings us one step closer to our goal, improving the awareness of our rare condition in the larger population.”
For its part, Catalyst offers a LEMS education and resource site called LEMS Aware.
Added Daly: “Together, we forge pathways of support and understanding for those affected by rare diseases, illuminating a brighter future for all.”
Creating an awareness day for LEMS is a huge win for our community. … This brings us one step closer to our goal, improving the awareness of our rare condition in the larger population.
Catalyst’s Firdapse can be used by prescribed patients to reduce LEMS symptoms, which include muscle weakness, fatigue, lack of reflexes, and speech, vision, and swallowing problems. The treatment was first granted U.S. approval in 2018 for patients ages 17 and older; that was expanded in 2022 to include children as young as age 6.
Firdapse had been cleared by the European Commission in 2009 for adult patients. The therapy also is approved in Canada for adults with LEMS.
The LEMS Family Association created the Awareness Day to seek to heighten the visibility of the autoimmune condition. Given its nonspecific signs and symptoms, it can be difficult to diagnose.
Further, because the disorder is widely unknown, patients may experience a dearth of understanding from family members or friends — and may lack support from clinicians, including specialists like neurologists.
The March 30 date was chosen to coincide with the 1957 publication of pioneering LEMS research conducted by the two physicians for whom the disease was named: Edward Lambert, MD, and Lee Eaton, MD, who first described the syndrome. According to Catalyst, their research “greatly advanced” the disorder’s understanding and treatment.
The nonprofit was pleased with the company’s support.
“We couldn’t be more thrilled to celebrate the milestone of this first LEMS Awareness Day together,” Wooldridge said.
The LEMS Family Association also offers a LEMS patient registry to collect patient data, with the aim of gaining a better understanding of the rare condition.
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