Researchers are looking for people with Lambert-Eaton myasthenic syndrome (LEMS) to participate in a phase 3 clinical trial, being conducted by Catalyst Pharmaceuticals, to test the investigational drug amifampridine phosphate. The Firdapse Strength Trial for LEMS, or FirST-4-LEMS, study is designed to evaluate the effectiveness of amifampridine phosphate in controlling, reducing and/or eliminating symptoms of . . .
CMT doesn’t hold Nicki back; it pushes her to reach higher. Literally. Read on to follow Nicki on the trek of a lifetime — to the top of Mount Kilimanjaro, the tallest mountain in Africa.
Researchers are looking for people with congenital myasthenic syndrome (CMS) to participate in a phase 3 study being conducted by Catalyst Pharmaceuticals to test the experimental drug amifampridine phosphate.
Amifampridine phosphate is a potassium channel inhibitor designed to cause greater stimulation of muscle by prolonging nerve signals and is expected to help treat muscle weakness in people with CMS. The drug has shown promising results in treating patients with a related disorder called LEMS.
For more than 60 years, firefighters have teamed with MDA to fight back against muscular dystrophy, ALS and related life-threatening diseases.
For one firefighter, the cause became very personal when his son, Chandler Mallen, was diagnosed with juvenile dermatomyositis, a neuromuscular disease that involves inflammation of the muscles. When Chandler asked his father — Scott, a firefighter who has collected donations for MDA’s Fill the Boot program for almost 25 years — if he’d lose the use of his legs, he didn’t wait for an answer.
“Even if I do,” Chandler said, “I’ll never stop trying.”
Osaka-based Mitsubishi Tanabe Pharma has reported encouraging 12-month efficacy and safety data for edaravone (brand name Radicava) for the treatment of ALS. Edaravone is delivered by intravenous injection. It’s thought to work by relieving the effects of oxidative stress, which, in people with ALS, has been suspected to play a role in the death of . . .
Results from the STEADFAST phase 3 clinical trial in Friedreich’s ataxia (FA) failed to show that the drug ACTIMMUNE® (interferon gamma-1b) was effective against any of the study’s disease measurements, according to Horizon Pharma. The study hoped to slow disease progression as measured by a functional rating scale assessing capacities such as speech, ability to . . .
Five Questions with Researcher Matthew Disney Matthew Disney, a professor at the Scripps Research Institute in Jupiter, Fla., was awarded an MDA research grant totaling $300,000 over a period of three years to optimize two novel drug-like compounds — one that targets toxic RNA in myotonic dystrophy and another that targets toxic RNA in ALS . . .
Researchers at the University of Kansas and Arkansas Children’s Hospital are looking for boys and men age 10 years and older with Duchenne muscular dystrophy (DMD) to participate in a phase 3 clinical trial. The study, called SIDEROS, is designed to help researchers determine the safety and efficacy of idebenone (brand name Raxone), an experimental . . .
Researchers are seeking volunteers to participate in a clinical trial sponsored by ReveraGen BioPharma to test the safety and effectiveness of the investigational drug vamorolone in boys with Duchenne muscular dystrophy (DMD). In the two-part, open-label study called “A Phase 2a and 2a Extension study to Assess Vamorolone in Boys with Duchenne Muscular Dystrophy,” all . . .
In a joint statement today, Biogen and Ionis Pharmaceuticals announced encouraging interim results from the late-stage CHERISH clinical trial to test the experimental drug nusinersen (brand name Spinraza) in children with type 2 spinal muscular atrophy (SMA). Based on the results, investigators have stopped the trial to allow all participants to transition into an open-label . . .
