CMS Study Seeks Participants

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.

Read More

ACTIMMUNE Phase 3 Trial in Friedreich’s Ataxia Disappoints

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 . . .

Read More

SIDEROS DMD Clinical Trial Seeks Participants

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 . . .

Read More

Participants Sought for Clinical Trial to Test Vamorolone in DMD

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 . . .

Read More

Encouraging Results in Nusinersen (Spinraza) Trial for Type 2 SMA

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 . . .

Read More

Biogen and Ionis Pharmaceuticals: A Message to the SMA Community

In a joint statement today, Biogen and Ionis Pharmaceuticals announced encouraging 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 trial . . .

Read More

FDA Will Review Nusinersen for Treatment of SMA

The U.S. Food and Drug Administration (FDA) has accepted Biogen’s New Drug Application for nusinersen (brand name Spinraza) for the treatment of spinal muscular atrophy (SMA) and granted Priority Review. Priority Review status is reserved for drugs that offer significant improvements over existing options or provide a treatment for a disorder for which no approved . . .

Read More