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Duchenne muscular dystrophy (DMD) is a severely debilitating childhood neuromuscular disease that affects 1 in 3,500 live male births. This rare disease is caused by mutations in the dystrophin gene...
The dystrophin gene is the largest gene in the body, consisting of 79 exons. Exons are small sequences of genetic code which, via an intermediate step involving RNA, lead to the assembly of sections...
Examples of Ophan Drug Indications include: Anaemia, sickle cell Cystic fibrosis (CF) Duchenne muscular dystrophy Glioma Graft vs host disease (GvHD) Hepatoma, liver cancer Hodgkin Lymphoma ...
Removing an immunomodulatory protein called osteopontin improves the symptoms of mice with muscular dystrophy by changing the type of macrophages acting on damaged muscle tissue, according to a paper ...
A group of Japanese scientists have succeeded in decoding a sugar molecule and clarifying a mechanism linked to muscular dystrophy. Their discovery has potential implications for muscular dystrophy tr...
The path to marketing an effective drug for Duchenne muscular dystrophy (DMD) hit another snag earlier this week.Read more about The State of the Muscular Dystrophy Drug PipelineComments
(Rockefeller University Press) Removing an immunomodulatory protein called osteopontin improves the symptoms of mice with muscular dystrophy by changing the type of macrophages acting on damaged muscl...
There is no effective drug approved for patients suffering from oculopharyngeal muscular dystrophy.
MILWAUKEE, Wis. (PRWEB) May 11, 2016 More than 1,300 business, community and civic leaders are putting their muscle behind raising c...
NICE has¬†recommended ataluren (Translarna, PTC Therapeutics) for treating children aged 5 and over with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation. The drug has been called a ‚Ä...
In a new study, a research team at Basel University Hospital in Switzerland investigates the biochemical and physiological characteristics of orbicularis oculi, a group of facial muscles that control ...
This study investigated quality of life (QOL) in adolescent and young men with Duchenne muscular dystrophy (DMD).
Severe intellectual disability has been reported in a subgroup of patients with Duchenne muscular dystrophy but is not typically associated with Becker muscular dystrophy.
To evaluate the diagnostic outcomes in a large cohort of congenital muscular dystrophy (CMD) patients using traditional and Next Generation Sequencing (NGS) technologies.
Duchenne muscular dystrophy (DMD), an X-linked disorder, is the most common muscular dystrophy with an incidence in boys of about 200 per million births. It presents in early childhood leading to deat...
Improved virological and immunological outcomes and reduced toxicity of antiretroviral combination therapy (ART) raise the hope that life expectancy of HIV-positive persons on ART will approach that o...