Dyne Therapeutics (DYN) last night announced additional one-year data from its ongoing Phase 1/2 ACHIEVE clinical trial of zeleciment basivarsen ...
Myotonia is a rare condition in which the muscles are unable to relax after they contract. This can affect muscles throughout the body and is caused by changes in the genetics of the individual, ...
Gamstorp specifically excluded clinical myotonia from her syndrome: "The typical inability to relax after a muscle contraction in myotonia congenita is not seen during or between attacks of adynamia ...
Today’s information comes to us from the NIH U.S. National Library of Medicine Genetics Home Reference. Myotonia congenita is a disorder that affects muscles used for movement. Beginning in childhood ...
Dyne will also present encore data on zeleciment rostudirsen (z-rostudirsen, formerly known as DYNE-251, aninvestigational medicine being evaluated for the treatment of individuals with DMD mutations ...
The study, published in Lancet Neurology, detailed the "head-to-head" trial implemented by the researchers to test two drugs, mexiletine and lamotrigine, on people with the condition. The trial, which ...
IN 1909, Steinert described a familial, chronic, slowly progressive disease characterized by visual and glandular disturbances combined with muscular atrophy. This has been called myotonia atrophica, ...
Investigators recently used a targeted drug to restore muscle strength and correct myotonia in mice with myotonic dystrophy. People with myotonic dystrophy experience progressive muscle weakness and ...
The purpose of this study was to characterize the entire coding region of the CLCN1 gene in a large cohort of Italian patients to define genetic epidemiology and identify population-specific genetic ...
New Rochelle, NY, February 21, 2013—By targeting the specific mutation that causes the hereditary neuromuscular disease myotonic dystrophy, it is possible to neutralize the mutant RNA toxicity and ...