In a joint statement today, Biogen and Ionis Pharmaceuticals announced that Biogen has completed the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of nusinersen, an investigational treatment for spinal muscular atrophy (SMA). Submission of the NDA is the first step toward getting approval . . .
Investigators are seeking people to participate in a phase 2 clinical trial, sponsored by Idera Pharmaceuticals, to evaluate the effect of the experimental drug IMO-8400 on skin lesions and muscle weakness in dermatomyositis. While the cause of dermatomyositis is unclear in most cases, it is known that ongoing inflammation triggered by the disease, over time, . . .
Researchers are looking for boys and men, ages 5 to 18 years, to participate in a natural history study that is designed to assess the potential of imaging techniques to monitor disease progression and serve as an outcome measure for clinical trials in Duchenne muscular dystrophy (DMD). Both healthy volunteers and individuals with DMD are . . .
In a joint statement today, Biogen and Ionis Pharmaceuticals announced encouraging interim results from the late-stage ENDEAR clinical trial to test the experimental drug nusinersen in infants with type 1 spinal muscular atrophy (SMA). Based on the results, investigators have stopped the trial to allow all participants to transition into an open-label trial called SHINE, . . .
BioMarin Pharmaceutical has announced that it is discontinuing development of BMN 701, an enzyme replacement therapy (ERT) for the treatment of Pompe disease (acid maltase deficiency, or AMD). In Pompe, a deficiency of an enzyme called acid alpha-glucosidase helps break down sugar stored in cells (glycogen). Without this enzyme, glycogen accumulates in cells and . . .
Marathon Pharmaceuticals announced today it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the investigational drug deflazacort for the treatment of people with Duchenne muscular dystrophy (DMD). The FDA has 60 days to determine whether the NDA is complete and acceptable for filing. Deflazacort, a glucocorticoid, works . . .
Results from a phase 3 trial to test eculizumab in patients with refractory generalized myasthenia gravis (MG) show that the drug did not have a statistically significant effect on the primary endpoint — the Myasthenia Gravis – Activities of Daily Living profile (MG-ADL), a patient-reported index of daily living assessment. Alexion Pharmaceuticals, which is . . .
BioMarin Pharmaceutical announced May 31 that it is discontinuing development of its exon skipping drug drisapersen. The company recently withdrew its Marketing Authorization Application (MAA) from the European Medicines Agency (EMA) following discussions at the May 2016 Committee for Medicinal Products for Human Use (CHMP) meeting that indicated the CHMP intended to issue a negative . . .
The investigational drug omigapil, under development by Santhera Pharmaceuticals, has received U.S. Food and Drug Administration (FDA) Fast Track designation for the treatment of congenital muscular dystrophy (CMD). Preclinical studies in disease models have shown the drug inhibits cell death and reduces body weight loss and skeletal deformation while increasing mobility and improving . . .
The Informed Consent Authorization Form is 12 pages long. My husband, Allen Carney, reads it carefully, initials in several places and signs at the end. And that’s the easiest part of participating in a research project. Welcome to the world of giving your time and body to research for Facioscapulohumeral muscular dystrophy. My husband Allen, . . .
