Researchers at Cytokinetics, Inc. are seeking adults living with amyotrophic lateral sclerosis (ALS) to participate in the phase 3 COURAGE-ALS clinical trial to evaluate the safety and effectiveness of the investigational drug. In ALS, motor neurons (nerve cells that control muscle cells) are gradually lost, causing the skeletal muscles they control to become weak and nonfunctional. . . .
On May 12, the US Food and Drug Administration (FDA) granted approval to an oral suspension of edaravone (RADICAVA ORS) for the treatment of amyotrophic lateral sclerosis (ALS). To date, RADICAVA ORS is the sixth disease modifying drug approved to treat ALS. RADICAVA ORS will be made available in the United States (US) and marketed . . .
It’s been half a century since University of California, Los Angeles, computer science professor Jacques Vidal coined the term “brain-computer interface” (BCI) and produced the first peer-reviewed papers on the idea of interpreting brain signals with a computer. Today, BCI offers huge potential to improve the lives of millions of people around the world, including . . .
Inclusion body myositis (IBM) is a rare, progressive muscle disease characterized by chronic muscle inflammation and weakness. It is estimated that approximately 20,000 people in the United States (US) have IBM, though the exact prevalence is unknown. IBM usually develops after age 50 and is more likely to affect men than women. The disease progresses . . .
On April 14, Edgewise Therapeutics announced the initiation of an Edgewise-funded observational trial in individuals with Becker muscular dystrophy (BMD). The goal of trial is to understand the progression of disease in individuals with BMD as assessed by functional measures and imaging endpoints. BMD is a genetic disease caused by a mutation in the dystrophin . . .
Drug companies are developing more therapies for rare disease patients — and doing it faster — than ever before. But when it comes to enrolling minorities in clinical trials, the US track record is lagging behind. That subject was the focus of a March 15 session during the 2022 MDA Clinical & Scientific Conference in . . .
On April 5, an international research team, headed by Michela Guglieri at Newcastle University and Robert Griggs at the University of Rochester Medical Center, announced positive results at the American Academy of Neurology (AAN meeting) in Seattle, WA from the phase 3 FOR DMD study of corticosteroid dosing regimens for treatment of people with Duchenne . . .
Researchers at Alexion Pharmaceuticals are seeking adults living with dermatomyositis to participate in a phase 2 clinical trial to evaluate the safety, effectiveness, and pharmacological properties of the investigational drug ravulizumab (Ultomiris). Ravulizumab is designed to target a component of the immune system (known as complement), which underlies many autoimmune disorders including dermatomyositis. Treatment with ravulizumab . . .
Myotonic dystrophy (DM) is a type of muscular dystrophy that affects about 1 in 8,000 people worldwide. The disease is known by several names, including Steinert disease, after the doctor that first described it, and “DM” in reference to its Greek name, dystrophia myotonica. Similar to other forms of muscular dystrophy, DM is characterized by . . .
When Yuliya Matyushenko was 12 years old, the Paris-based rare disease organization Eurordis featured her on a poster promoting Rare Disease Day 2015. In late February, the young woman with spinal muscular atrophy (SMA) was hiding in a bare concrete basement in Kharkiv as Ukraine’s second-largest city, located only 35 kilometers from the Russian border, . . .