In recognition of International Day of Women and Girls in Science, the Muscular Dystrophy Association (MDA) is honored and excited to highlight the career and accomplishments of Dr. Angela Lek, PhD. International Women and Girls in Science Day, February 11, endeavors to acknowledge and celebrate the invaluable role that women and girls play in accelerating . . .
Becker muscular dystrophy (BMD) is a rare genetic disorder involving mutations of the dystrophin gene. BMD exhibits similar signs and symptoms to another condition, Duchenne muscular dystrophy (DMD), which is caused by different mutations in the same gene. Although both conditions affect skeletal muscles used for movement, as well as heart muscles, they differ in . . .
On Dec. 17, the US Food and Drug Administration (FDA) granted approval to efgartigimod (Vyvgart) for the treatment of generalized myasthenia gravis (gMG) in adults who test positive for the anti-acetylcholine receptor (AChR) antibody. To date, Vyvgart is the second disease modifying drug approved to treat gMG. Vyvgart will be made available in the United States . . .
Researchers at Boston Children’s Hospital/Brandeis University are seeking unaffected siblings of boys with Duchenne Muscular Dystrophy (DMD) to participate in a survey about their perspectives on disclosure and communication within the family. The information gained from this study will be used to guide parents on how to best communicate information about DMD to their family members. . . .
Researchers at Worrell are seeking adolescents (ages 12 – 17) with the following conditions: neuromyelitis optica spectrum disorder (NMOSD), myasthenia gravis (gMG), myelin oligodendrocyte glycoprotein antibody disease (MOG-AD), or autoimmune encephalitis (AE), to participate in a research study to test the safety and efficacy of a new syringe device being developed to administer a drug to . . .
Researchers at the Hospital for Sick Children in Toronto, Canada are seeking young adults living with muscular dystrophy to participate in a survey about their interest in and experiences receiving genetic information. Participants will be required to complete a 10-15 minute online survey that collects largely quantitative data, as well as some open-ended responses. After completing . . .
Researchers at the University of Florida are seeking adult males living with Becker muscular dystrophy (BMD) to participate in a three-year natural history study. The objective of this observational study is to gain a better understanding of the course of BMD and to identify biomarkers for BMD. This study does not involve an intervention. Enrolled participants will . . .
Researchers at AMO Pharma Ltd are seeking children and adolescents living with congenital myotonic dystrophy (DM1), also known as Steinert’s disease, to participate in a phase 2/3 clinical trial (REACH CDM) to evaluate efficacy of the investigational drug tideglusib (AMO-02) to treat congenital DM1. Tideglusib is designed to disrupt the RNA repeat that causes congenital DM1, . . .
Researchers at Stanford Neuroscience Health Center are seeking 3-year-old boys living with Duchenne muscular dystrophy (DMD), particularly in California, to participate in an early phase clinical trial (a new cohort of the ENDEAVOR trial) to evaluate efficacy of Sarepta’s investigational gene replacement therapy SRP-9001 to treat DMD. SRP-9001 uses an adeno-associated virus (AAVrh74) to introduce a . . .
On Oct. 28, 2021 the Muscular Dystrophy Association (MDA) announced the awarding of 18 new grants totaling over $1.6 million toward neuromuscular disease (NMD) research. These new grants represent a continued commitment by MDA to fund groundbreaking research that will one day lead to treatments and cures for the diseases in its program. The newly . . .