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Latest Limb-girdle muscular dystrophy Stories

2014-09-10 12:28:50

However, Less Than One-Third of Duchenne Patients Are in Subpopulations That Will Benefit From New Disease-Modifying Therapies, According to Findings from Decision Resources Group BURLINGTON, Mass., Sept. 10, 2014 /PRNewswire/ -- Decision Resources Group finds that the first three emerging disease-modifying therapies (DMTs)--PTC Therapeutics' Translarna (ataluren), Prosensa's drisapersen and Sarepta's eteplirsen--will likely receive regulatory approval for Duchenne muscular dystrophy (DMD) in...

2013-09-18 12:55:59

A preclinical study led by researchers at Children’s National Medical Center has found that a new oral drug shows early promise for the treatment of Duchenne muscular dystrophy (DMD). The results, published in EMBO Molecular Medicine, show that the drug, VBP15, decreases inflammation and protects and strengthens muscle without the harsh side effects linked to current treatments with glucocorticoids such as prednisone. Duchenne muscular dystrophy results in severe muscle degeneration and...

2013-08-09 09:43:14

Study identifies 3 enzymes required for building sugar superstructure involved in muscular dystrophies For many inherited diseases, such as cystic fibrosis or Huntington disease, the disease-causing genetic mutation damages or removes a protein that has an essential role in the body. This protein defect is the root cause of the disease symptoms. However, for a group of muscular dystrophies known collectively as congenital muscular dystrophies (CMDs), the sequence of the protein that is...

Experimental Gene Therapy Treatment Offers Hope For Youngster With Duchenne Muscular Dystrophy
2013-02-09 09:05:38

UC Davis Health System [ Watch The Video ] Jacob Rutt is a bright 11-year-old who likes to draw detailed maps in his spare time. But the budding geographer has a hard time with physical skills most children take for granted “• running and climbing trees are beyond him, and even walking can be difficult. He was diagnosed with a form of muscular dystrophy known as Duchenne when he was two years old. The disease affects about 1 in 3,500 newborns “• mostly boys...

2012-07-09 10:54:16

The challenge of treating patients with genetic disorders in which a single mutated gene is simply too large to be replaced using traditional gene therapy techniques may soon be a thing of the past. A Nationwide Children's Hospital study describes a new gene therapy approach capable of delivering full-length versions of large genes and improving skeletal muscle function. The strategy may hold new hope for treating dysferlinopathies and other muscular dystrophies. A group of untreatable...

2012-06-28 06:28:07

(Ivanhoe Newswire) - Over 3,000 Americans are affected by muscular dystrophy, a genetic disorder primarily affecting skeletal muscle that results in greatly impaired mobility and, in sever cases, respiratory and cardiac dysfunction. For the first time, scientists have turned muscular dystrophy patients' fibroblast cells (common cells found in connective tissue) into stem cells and then differentiated them into muscle precursor cells. The muscle cells were then genetically modified and...


Word of the Day
caparison
  • A cloth or covering, more or less ornamented, laid over the saddle or furniture of a horse, especially of a sumpter-horse or horse of state.
  • Clothing, especially sumptuous clothing; equipment; outfit.
  • To cover with a caparison, as a horse.
  • To dress sumptuously; adorn with rich dress.
This word ultimately comes from the Medieval Latin 'cappa,' cloak.
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