A therapeutic strategy that combines gene therapy and stem cell transplantation has shown encouraging results in mice with a disorder mimicking Duchenne muscular dystrophy (DMD).
R. Rodney Howell — respected geneticist, pediatrician and chairman of the MDA Board of Directors — has been honored by the March of Dimes for playing a key role in developing the uniform panel of serious disorders for which nearly every baby in the United States now is tested.
Proposals exploring the feasibility and advisability of implementating newborn screening for two disorders in MDA's program — Duchenne muscular dystrophy and
Spinal muscular atrophy (SMA) is a disease in which nerve cells that control muscles (motor neurons) in the spinal cord die, causing progressive weakness in the voluntary muscles.
An international patientregistry (database) is gathering information about children and adults with any form of congenital muscular dystrophy (CMD), a congenital my
The number of new cases of amyotrophic lateral sclerosis (ALS) identified each year (incidence) and the number of people living with the disease (prevalence) appears to be lower in American Indians and Alaska Natives than in white populations.
As scientists learn more about what our DNA can tell us about health and disease, public interest has intensified and genetic testing has become increasingly common.
Mice with a disorder mimicking human spinal-bulbar muscular atrophy(SBMA, or Kennedy disease) that were treated with an experimental therapy called arimoclomol showed improved nerve-cell survival, increased body weight, and better muscle strength and functi
Scientists at the biopharmaceutical company Genzyme, working with mice, say they have modified and improved an existing experimental strategy to treat type 1 myotonic muscular dystrophy (MMD1, also known as DM1).
In the United States, a disease or disorder is defined as “rare” when it affects fewer than 200,000 Americans at any given time; in Europe, when it affects less than one in 2,000 people.
The U.S. Food and Drug Administration (FDA) will host a public hearing Monday, Feb. 25, 2013, from 9 a.m. to 5 p.m. Eastern time, in Silver Spring, Md., to discuss the development of drugs for amyotrophic lateral sclerosis (ALS).
Trials of the experimental muscular dystrophy drug ataluren showed that the drug was generally well-tolerated and slowed the rate of decline in walking ability, compared to the placebo group.
The experimental drug AT2220 has shown benefit as an enhancer of enzyme replacement therapy for the metabolic muscle disorder Pompe disease (acid maltase deficiency).
Prednisone and other drugs in the corticosteroid family have become standard treatment for Duchenne muscular dystrophy (DMD) in much of the world for almost a decade. In this disorder, they slow the decline of muscle function and prolong walking ability.