April 12, 2010
The experimental drug Zenvia, developed by Avanir Pharmaceuticals as a treatment for unwanted laughing/crying spells in ALS, has shown continued promise in lessening the frequency of such episodes
April 12, 2010
The experimental drug Zenvia, developed by Avanir Pharmaceuticals as a treatment for unwanted laughing/crying spells in ALS, has shown continued promise in lessening the frequency of such episodes in
April 12, 2010
An MDA-supported, 10-center trial of more than 100 people with amyotrophic lateral sclerosis (ALS) who took the oral medication lithium carbonate — some in conjunction with the ALS drug riluzole —
April 05, 2010
MDA has awarded two grants for research aimed at determining the precise molecular causes of facioscapulohumeral dystrophy (FSHD), and developing therapies for the disease.
MDA and Friends of FSH
April 02, 2010
The state of research in the familial (inherited) form of ALS was the focus of a special one-hour Webinar presented by the ALS Therapy Development Institute (ALS TDI) on March 25, with reports by CEO
April 02, 2010
The state of research in the familial (inherited) form of ALS was the focus of a special one-hour Webinar presented by the ALS Therapy Development Institute (ALS TDI) on March 25, with reports by
March 29, 2010
Blocking a key molecular pathway that the body uses to amplify an immune response has been found to delay disease onset and extend survival in mice with a disease that mimics human amyotrophic
March 28, 2010
Blocking a key molecular pathway that the body uses to amplify an immune response has been found to delay disease onset and extend survival in mice with a disease that mimics human amyotrophic
March 24, 2010
Update (Oct. 8, 2012): This story has been updated to reflect that the tadalafil trial in BMD has been slightly revised and is now open. In June 2012, it had temporarily closed to new participants.
March 23, 2010
An MDA-supported clinical trial of intravenous gentamicin in children and adolescents with a form of Duchenne muscular dystrophy (DMD) caused by so-called "nonsense" mutations (also called "premature
March 22, 2010
A synthetic compound that seals cellular membranes has been found to stop the progression of heart-muscle destruction in dogs with a disease closely resembling human Duchenne muscular dystrophy (DMD
March 17, 2010
A research team at the University of Pennsylvania in Philadelphia has characterized the mechanism responsible for rapid decay of the survival of motor neurons (SMN) protein that is encoded by the
March 08, 2010
MDA grantee Dongsheng Duan has published a book on the latest advances in gene therapy for muscle disease, particularly muscular dystrophies.
Duan, a professor of microbiology and immunology at the
March 05, 2010
Isis Pharmaceuticals of Carlsbad, Calif., has begun a phase 1 clinical trial of its experimental compound ISIS-SOD1-Rx in people with familial (inherited) ALS caused by toxic SOD1 protein molecules
March 05, 2010
Isis Pharmaceuticals of Carlsbad, Calif., has begun a phase 1 clinical trial of its experimental compound ISIS-SOD1-Rx in people with familial (inherited) ALS caused by toxic SOD1 protein molecules
March 03, 2010
The biopharmaceutical firm PTC Therapeutics announced March 3 that ataluren, its experimental drug for certain forms of Duchenne (DMD) and Becker (BMD) muscular dystrophy, although safe and well
March 01, 2010
Scientists at four U.S. institutions have successfully used gene therapy to treat very young mice with a disease resembling severe spinal muscular atrophy (SMA). Study results were published online
February 26, 2010
A process called trans-splicing has been shown to increase levels of a needed protein in mice with a disease resembling severe human spinal muscular atrophy (SMA), says a research team at the
February 26, 2010
The National Institutes of Health (NIH) and the U.S. Food and Drug Administration (FDA) have announced plans to establish a Joint NIH-FDA Leadership Council, with the goal of shortening the time it
February 23, 2010
Remodeling a building usually requires some degree of dismantling before new construction can begin. That principle, it now seems, also may apply to the remodeling of the body's cells.
Scientists at
February 22, 2010
MDA-supported researchers at the University of Ottawa and Ottawa Hospital Research Institute (OHRI) have identified a biological pathway that may prove useful in developing treatments for spinal
February 18, 2010
An MDA-supported, multinational team of researchers from Canada and Europe has identified specific mutations in the anoctamin 5 (ANO5) gene on chromosome 11 that can cause type 2L limb-girdle
February 12, 2010
This story was updated Oct. 6, 2010.
In many forms of muscular dystrophy, including Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), weakness and degeneration of the cardiac
February 09, 2010
MDA-supported scientists in France have identified a previously unknown type of muscle stem cell located in the spaces between muscle fibers in mice. They say the new cells, dubbed "PICs," may play
February 02, 2010
Scientists at several U.S. institutions have added yet another piece of the puzzle of type 1 myotonic dystrophy (MMD1, also called DM1).
Much of the problem in this disease appears to be caused by