Rx] for the treatment of myotonic
dystrophy type I.
dystrophy--the most common adult form of muscular dystrophy--is a chronic, progressive, and inherited neuromuscular condition presenting with multisystemic complications including distal muscle weakness, myotonia, hypersomnolence, early-onset cataracts, cardiac conduction abnormalities, and slurred speech and swallowing problems (Harper, 2001).
AMO Pharma is currently advancing two investigational therapies for treatment of fragile X syndrome and myotonic
dystrophy, and is in the process of licensing additional development-stage products in diverse areas including autism and other CNS disorders.
Both types of myotonic
dystrophy are inherited disorders that involve progressive muscle wasting and weakness, and both are caused by a type of genetic defect known as a "RNA repeat expansion," a series of nucleotides repeated more times than normal in an individual's genetic code.
In the new study, Disney and Research Associate Lirui Guan attached a rationally designed small molecule that targets the RNA that causes myotonic
dystrophy type 1 with a molecule that produces hydroxyl radicals.
A 24-year-old man with myotonic
dystrophy type 1 was referred to our division for dyspnea and palpitations.
muscular dystrophy affects about 35,000 people in the U.
The use of sugammadex to reverse rocuronium in a patient with myotonic
Vernon was the sole carer for his wife, who suffered from the muscle-wasting disease myotonic
At that time, the FDA said the vast majority of the 4 million prescriptions written for quinine sulfate each year were for off-label uses, including nocturnal leg cramps, restless leg syndrome, and other myotonic
Iplex, which is already approved for treating a growth hormone deficiency, was being tested by the company as a potential treatment for myotonic
muscular dystrophy (MMD), a genetic disorder that results in various symptoms across multiple body systems.
Heading this collection are short communications on considering myotonic
dystrophy as a "repeat disease" and using one or two individual organisms to address population-wide genetic questions.