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Duchenne muscular dystrophy is a rare progressive disease that eventually affects all voluntary muscles and involves the heart and breathing muscles in later stages. Life expectancy is estimated to be around 25–26, [18] [59] but this varies. People born with Duchenne muscular dystrophy after 1990 have a median life expectancy of approximately ...
Many affected people will eventually become unable to walk [2] and Duchenne muscular dystrophy in particular is associated with shortened life expectancy. Muscular dystrophy was first described in the 1830s by Charles Bell. [2] The word "dystrophy" comes from the Greek dys, meaning "no, un-" and troph-meaning "nourish". [2]
Dystrophinopathy refers to a spectrum of diseases due to mutations in the DMD gene, which encodes for the dystrophin protein found in muscle. [1] [2] [3] The severe end of the spectrum includes Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD), and DMD-associated dilated cardiomyopathy.
(Reuters) -A young patient died due to cardiac arrest after receiving Pfizer's experimental gene therapy being tested in a mid-stage trial for a muscle-wasting disorder called Duchenne muscular ...
Darius Goes West: The Roll of his Life is a documentary film by Logan Smalley about Darius Weems, a teenager living with Duchenne muscular dystrophy.In the middle of 2005 Weems embarked on a 7,000 mile road trip across the United States from his hometown in Georgia to MTV Headquarters in Los Angeles to ask them to customize his wheelchair on Pimp My Ride, as well to promote awareness of the ...
Shares of Prosensa , a biotech company developing the drug drisapersen for the treatment of Duchenne muscular dystrophy, or DMD), plunged 70% today after the company revealed that the drug failed ...
The gene affected is the DMD gene, is located on the X chromosome and is inherited in an X-linked recessive pattern. [13] Since women have two X chromosomes, if one X chromosome has the non-working gene, the second X chromosome will have a working copy of the gene to compensate, because of this ability to compensate, women rarely develop symptoms.
Variants in the DMD gene that lead to the production of too little or a defective, internally shortened but partially functional dystrophin protein, result in a display of a much milder dystrophic phenotype in affected patients, resulting in the disease known as Becker's muscular dystrophy (BMD). In some cases, the patient's phenotype is such ...