Scholar Rock Holding Corporation announced positive results from its phase 1 clinical trial evaluating SRK-015, a therapy for treating spinal muscular atrophy (SMA), at the Cure SMA Annual Conference held June 28 through July 1 this year. Consistent with previously announced interim findings in February, the final results showed positive safety, pharmacodynamic, and pharmacokinetic data in healthy volunteers, supporting . . .
The muscular dystrophies are a group of neuromuscular diseases that cause weakness and degeneration of the skeletal (voluntary) muscles. Muscular dystrophies are generally considered genetic diseases, or diseases that arise from genetic mutations, which can be either inherited or spontaneous. Some muscular dystrophies have been found to have a genetic component, which means a genetic . . .
MDA has awarded MDA Venture Philanthropy (MVP) funding totaling $389,463 over two years to Rachelle H. Crosbie, PhD, professor and chair of Integrative Biology and Physiology at the University of California, Los Angeles. The award will support the development of a small molecule drug that increases expression of sarcospan, a protein that may help to . . .
At the 2019 MDA Clinical & Scientific Conference, one scientific session of interest centered on what’s new in neuromuscular diseases (NMDs). In this session, presenting scientists discussed progress in therapy development and clinical trials for a range of NMDs, including Becker muscular dystrophy (BMD), congenital muscular dystrophy (CMD), congenital myasthenic syndromes (CMS), myotonic dystrophy (DM), . . .
The results of the Christopher Project have been published in a comprehensive, 80-page report. The creation of “The Christopher Project Report to the Myotonic Dystrophy Community” was supported by several organizations, including the Muscular Dystrophy Association (MDA), and is available for download on MDA’s website. (Printed copies are also available upon request.) What is the . . .
Myasthenia gravis (MG) is an autoimmune disease, which is a disease that occurs when the immune system goes awry and produces antibodies that attack the body’s own tissues. Normally, antibodies are created to rid the body of pathogens such as bacteria, viruses, and foreign substances. In MG, antibodies attack the connection between nerve and muscle . . .
Adam Bittel, PT, DPT, PhD, a postdoctoral fellow at Children’s National Medical Center in Washington, DC, was awarded the 2019 SSSI-MDA Fellowship Award. The award, co-sponsored by Strength, Science & Stories of Inspiration (SSSI) and MDA, will provide a total of $40,000 over two years to support Dr. Bittel’s work investigating the cellular mechanisms underlying the effects . . .
Like many pre-medical students, Justin Cohen discovered along the way that what he really liked was research. However, unlike others who exchange the stethoscope for a microscope, Justin had a strikingly different motivating factor — he has been living with the disease he studies, facioscapulohumeral muscular dystrophy (FSHD), for almost as long as he can . . .
Today, MDA and Locana, a leading RNA-targeting gene therapy company, announced the award of an MDA Venture Philanthropy (MVP) grant totaling $550,000 to advance Locana’s development program for myotonic dystrophy (DM), the most common form of adult-onset muscular dystrophy. MVP is the MDA’s drug development program that is exclusively focused on funding the discovery and . . .
On June 7, the U.S. Food and Drug Administration (FDA) approved Emflaza (deflazacort) to expand its labeling to include patients with Duchenne muscular dystrophy (DMD) who are between 2 and 5 years old. Emflaza was approved by the FDA in February 2017 for the treatment of DMD in patients 5 years and older, making it the first drug approved . . .