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  • noun

Words related to myopathy

any pathology of the muscles that is not attributable to nerve dysfunction

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Today, Paley is a bright, happy, deeply-loved five-year-old living with Myotubular Myopathy.
* Myotubular myopathy. This presents similarly, but the mother has no abnormalities.
Symptoms: X-linked myotubular myopathy usually affects only boys, and causes severe muscle weakness and hypotonia noticeable at birth and sometimes before.
Audentes Therapeutics provided an update following the receipt of minutes from its Regenerative Medicine and Advanced Therapy/Type B meeting with the FDA held in December regarding its lead gene therapy candidate, AT132, for the treatment of X-linked Myotubular Myopathy, or XLMTM.
a biotechnology company focused on developing and commercializing gene therapy products for patients living with serious, life-threatening rare diseases, today announced it has commenced dosing of patients in ASPIRO, a Phase 1 / 2 clinical trial of AT132 for the treatment of X-Linked Myotubular Myopathy (XLMTM).
The company has four product candidates in development, AT132 for the treatment of X-Linked Myotubular Myopathy (XLMTM), AT342 for the treatment of Crigler-Najjar Syndrome, AT982 for the treatment of Pompe disease, and AT307 for the treatment of the CASQ2 subtype of Catecholaminergic Polymorphic Ventricular Tachycardia (CASQ2-CPVT).
The infant suffers from Myotubular Myopathy - an rare illness which affects the muscles that control breathing and swallowing.
The severe form of myotubular myopathy also affects William's breathing and swallowing, meaning he requires round-the-clock care to survive.
Liam Barker suffers from myotubular myopathy, a muscle wasting disorder which means he can make only very small movements.
The infant has never known a life outside a hospital ward because he has a rare genetic disorder called Myotubular Myopathy.
At least one myopathy (a type of myotubular myopathy) is caused by mutations in a muscle protein required for normal muscle development.
He believes the Q3 of 2019 update from additional patients is likely to establish AT132 "not only as the first but best in class gene therapy" for X-linked myotubular myopathy. The analyst keeps a Buy rating on Audentes Therapeutics with a $33 price target.