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Latest Spinal muscular atrophy Stories

2011-10-05 19:32:27

Findings reveal that deficiency of the SMN protein in peripheral tissues might also contribute to SMA pathology A new study from Cold Spring Harbor Laboratory (CSHL) reports surprising results that suggest that the devastating neuromuscular disease, spinal muscular atrophy (SMA), might not exclusively affect the motor neurons in the spinal cord as has long been thought. The new findings suggest that defects in peripheral tissues such as liver, muscle, heart, etc., might also contribute to...

2011-09-27 11:31:29

Discovery could lead to treatments for muscular dystrophy and ALS Researchers at the University of Missouri have identified a communication breakdown between nerves and muscles in mice that may provide new insight into the debilitating and fatal human disease known as spinal muscular atrophy (SMA). “Critical communication occurs at the point where nerves and muscles ℠talk´ to each other. When this communication between nerves and muscles is disrupted, muscles do not...

2011-07-25 23:28:09

'Biggest increase anyone has seen' in the production of essential compound for deadly childhood disease Researchers in Ottawa report new hope for the treatment of infants born with serious genetic disorder. Over 1000 children in Canada are affected with Spinal Muscular Atrophy (SMA), a genetic disorder that causes muscle weakness and loss of motor control. In its most severe form survival of children with SMA beyond 5 years is rare. Although the disorder is caused by the loss of a specific...

2011-07-25 23:27:04

Spinal muscular atrophy (SMA) is the most frequently inherited cause of infant mortality. Two independent research groups "” one led by Alex MacKenzie, at Children's Hospital of Eastern Ontario Research Institute, Ottawa; and one led by Umrao R. Monani, at Columbia University Medical Center, New York, and Cathleen M. Lutz, at The Jackson Laboratory, Bar Harbor "” have now generated new data in mouse models of severe SMA that provide hope that a therapeutic providing meaningful...

2011-06-06 14:00:00

Failure in nerve-fiber navigation corrected in zebrafish model; suggests possibility of a drug treatment BOSTON, June 6, 2011 /PRNewswire-USNewswire/ -- Spinal muscular atrophy (SMA) is the leading genetic cause of death in children under 2, with no treatment other than supportive care. In the Proceedings of the National Academy of Sciences (Early Edition, week of June 6), researchers at Children's Hospital Boston show how loss or mutation of the SMA gene causes progressive muscle...

2011-05-19 07:37:00

First ever clinical trial approved for SMA with a novel drug specifically designed to treat the disease CHICAGO, May 19, 2011 /PRNewswire-USNewswire/ -- Families of Spinal Muscular Atrophy announced today that Repligen Corporation has received approval from the U.S. Food and Drug Administration to commence a Phase I safety study in healthy volunteers for RG3039, formerly called Quinazoline495, which is being developed for Spinal Muscular Atrophy. FSMA began the Quinazoline program in...

2011-05-04 09:48:00

WASHINGTON, May 4, 2011 /PRNewswire/ -- The 2011 FightSMA Annual Conference, the Good Fight, is underway at the L'Enfant Plaza Hotel this week, and experts in the fields of pulmonology, nutrition, orthopedics, clinical trial and neurology are gearing up to participate in the Thriving with SMA: LIVE! webcast on Thursday, May 5, 2011. This one-of-a-kind, unique web-based program is free to the public and will feature panel discussions by leaders in the SMA community. Online attendees and...

2011-04-29 15:22:00

WASHINGTON, April 29, 2011 /PRNewswire/ -- International non-profit organization FightSMA will be hosting highly respected experts from across the country on Thursday, May 5, 2011 for a series of panel discussions, Thriving with SMA: LIVE! This panel is a unique, one-of-a-kind series of presentations from influential leaders in the fields of pulmonary issues, orthopedics, nutrition, neurology and clinical trial. FightSMA will be joined by families, friends, healthcare professionals and...

2011-04-28 14:47:00

WASHINGTON, April 28, 2011 /PRNewswire/ -- FightSMA will be welcoming elite researchers and scientific professionals, as well as families and friends in the SMA community, to the L'Enfant Plaza Hotel May 3-6, 2011 in Washington, D.C. The group, which was founded in 1991, is celebrating its 20th anniversary this year. To date, FightSMA has raised more than $7 million for SMA research and boasts 19 chapters in the U.S. and Canada. The centerpiece of the Conference will be the Thriving with...


Word of the Day
Cthulhu
  • A gigantic fictional humanoid alien god being described with a head resembling an octopus and dragon wings and claws, around whom an insane cult developed.
  • Pertaining to the mythos of Cthulhu and additional otherworldly beings created by H. P. Lovecraft or inspired by his writings and imitators.
This word was invented in 1926 by H.P. Lovecraft for his short story, 'The Call of Cthulhu.' 'Cthulhu' may be based on the word 'chthonic,' which in Greek mythology refers to the underworld.
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