“The approval today of KYGEVVI® (doxecitine and doxribtimine), provides real hope for patients and families living with this devastating disease,” said Angela Lek, PhD, interim Chief Research Officer ...
Oculopharyngeal muscular dystrophy (OPMD) is a rare inherited disorder that causes weakness in the eye and throat muscles. It may lead to drooping eyelids and swallowing difficulties. Muscular ...
FDA Approves KYGEVVI® (doxecitine and doxribtimine), the First and Only Treatment for Adults and Children Living with Thymidine Kinase 2 deficiency (TK2d), a Rare and Devastating Mitochondrial Disease ...
Discover the various types, causes, symptoms, diagnosis, and treatments for muscular dystrophy, along with early warning signs and effective care strategies ...
Congenital muscular dystrophy (CMD) is a disease that affects certain muscles. Individuals with CMD may experience symptoms such as muscle weakness and joint problems. “Congenital” means present since ...
A regularly prescribed class of heart medications might be capable of treating one of the most common forms of muscular dystrophy, a new study in mice suggests. Myotonic dystrophy type 1 (DM1) is ...
Editor’s note: This is an automatically generated transcript. Please notify [email protected] if there are concerns regarding accuracy of the transcription. Patient advocacy organizations often bring ...
Adults with Duchenne muscular dystrophy demonstrated functional improvements exceeding natural history, following 28-day treatment with SAT-3247 Grip strength increased 118.6% and predicted forced ...
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