Tag : Genetic Disorder

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Wayne State researchers receive grant to develop new treatments for Barth syndrome

Newsemia
Barth syndrome is a rare and life-threatening, X-linked genetic disorder that primarily affects males and is passed from mother to son; women who are carriers...
Neurology

Maybe Memorizing the Krebs Cycle Was Worthwhile After All

Newsemia
Mitochondrial disorders are often difficult to diagnose and treat due to their multisystem nature. As scientists better understand mitochondrial function, effective treatments are beginning to...
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Children's National receives new funding to strengthen global polycystic kidney disease initiative

Newsemia
Lisa M. Guay-Woodford, M.D., McGehee Joyce Professor of Pediatrics at Children’s National Hospital, has received a $5.7 million dollar gift to support clinical and research...
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New discovery could improve therapies for Duchenne muscular dystrophy

Newsemia
A new multi-institution study spearheaded by researchers at Florida State University and the University of California, Los Angeles suggests a tiny protein could play a...
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New gene therapy cures babies with fatal 'Bubble Boy' disease

Newsemia
A new gene therapy created by US researchers appears to have cured eight infant boys born with a rare genetic disorder referred to as “Bubble...
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Scientists aim to find genetic causes of developmental abnormalities in the vagina and uterus

Newsemia
A percentage of females are born with a missing or underdeveloped vagina and uterus, and scientists are working to analyze the genes of hundreds of...
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Leadiant Biosciences announces FDA approval of Revcovi for treating pediatric, adult ADA-SCID patients

Newsemia
Leadiant Biosciences, Inc. today announced that the Food and Drug Administration has granted approval to Revcovi (elapegademase-lvlr) injection in the U.S. Revcovi is a new...
Neurology

ATTR-ACT: Tafamidis Reduces Mortality in Amyloid Cardiomyopathy

Newsemia
A drug that binds transthyretin reduced all-cause mortality and cardiovascular hospitalizations and slowed decline in functional capacity in patients with transthyretin amyloid cardiomyopathy. Medscape Medical...
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UC San Diego researchers awarded two grants for investigating stem cell-based therapies

Newsemia
The governing board of the California Institute for Regenerative Medicine unanimously approved this week two grants worth more than $2.2 million to University of California...

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