Brain-Computer Interface Technology Could Improve Life for Millions

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 . . .

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FDA Approves Alexion’s Ultomiris for Treatment of gMG

On April 28, the US Food and Drug Administration (FDA) granted approval to ravulizumab (Ultomiris) for the treatment of generalized myasthenia gravis (gMG) in adults who test positive for the anti-acetylcholine receptor (AChR) antibody. To date, Ultomiris is the third disease modifying drug approved to treat gMG. Ultomiris will be made available in the United . . .

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Simply Stated: Inclusion Body Myositis, Revisited

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 . . .

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Capricor Announces Positive Results from its Phase 2 Study of CAP-1002 to Treat DMD

On September 24, Capricor Therapeutics announced positive results at the World Muscle Society Virtual Congress from its phase 2 HOPE-2 trial of the investigational therapy CAP-1002 for treatment of people in advanced stages of Duchenne muscular dystrophy (DMD). The primary and secondary endpoints of the study, the improvement of upper limb and cardiac function, were met, . . .

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FDA Accepts Sarepta Therapeutics’ New Drug Application for DMD Treatment Casimersen

On Aug. 25, Sarepta Therapeutics announced that the US Food and Drug Administration (FDA) accepted the company’s New Drug Application (NDA) seeking accelerated approval for casimersen (SRP-4045), an investigational therapy for Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping. The FDA has granted Sarepta’s application Priority Review Status, meaning that a decision is expected . . .

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FDA Approves NS Pharma’s Viltepso for Treatment of DMD Amenable to Exon 53 Skipping

Today, the US Food and Drug Administration (FDA) granted accelerated approval to viltolarsen (Viltepso) for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 53. It is the third exon-skipping, disease-modifying drug to treat DMD, the most common childhood form of muscular dystrophy. The drug is administered by intravenous infusion. Viltepso will . . .

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FDA Approves Genentech’s Evrysdi for Treatment of SMA in Pediatric and Adult Patients

On Aug. 7, the US Food and Drug Administration (FDA) granted approval of risdiplam (Evrysdi) for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. It is the third disease-modifying therapy approved to treat SMA, the leading genetic cause of infant death. The drug is an orally administered liquid, the first medicine that could . . .

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How MDA is Supporting LGMD Research and Care in 2020

On July 18, MDA is hosting a free virtual Engage Limb-Girdle Muscular Dystrophy Symposium, a half-day educational event featuring key experts who will speak on topics including the latest in genetics and research in limb-girdle muscular dystrophy (LGMD). MDA has a longstanding commitment to finding treatments for the more than 30 subtypes of LGMD; since . . .

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Sarepta Therapeutics Announces Positive Results from the SRP-9001 Gene Therapy Trial to Treat DMD

On June 15, Sarepta Therapeutics announced positive results from its phase 1/2a study of SRP-9001 gene therapy to treat Duchenne muscular dystrophy (DMD). Data from four patients indicated that a single intravenous infusion of SRP-9001 was safe and well tolerated, with no serious adverse events. Additionally, all participants demonstrated improvements across multiple efficacy-related endpoints, including . . .

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