FDA grants accelerated approval for first treatment for Barth syndrome
The U.S. Food and Drug Administration has granted accelerated approval to Forzinity (elamipretide) injection as the first treatment for Barth syndrome.
Sep 25, 2025
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Cardiovascular Diseases: News and Research on Heart Defects, Congenital
MeSH tree: C14.280.400
Uncovering drivers of—and possible treatment for—Noonan syndrome heart defects
Noonan syndrome with multiple lentigines (NSML) is a rare genetic disorder that causes short stature, distinctive facial features, and clusters of dark skin spots called lentigines. But its most serious impact is a dangerous ...
Sep 16, 2025
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How a surgical team repaired a rare variation of D-transposition of the great arteries
When a critically ill baby with dextro-transposition of the great arteries (D-TGA) was transferred to Children's Hospital Los Angeles last year—just hours after birth—the Heart Institute team saw right away that the case ...
Sep 10, 2025
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Precision genetic target provides hope for Barth syndrome treatment
Researchers at The Hospital for Sick Children (SickKids) have uncovered a promising new therapeutic target for Barth syndrome, a rare genetic condition with no current cure.
Sep 3, 2025
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A cheek swab test could identify children with potentially deadly heart condition
A simple cheek swab test could detect children who have arrhythmogenic cardiomyopathy, according to research presented at the European Society of Cardiology's annual congress in Madrid.
Sep 1, 2025
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Living heart valves show promise across multiple pediatric conditions
A procedure which uses living heart valves may have expanded application as a treatment for various types of pediatric heart valve conditions.
Aug 27, 2025
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Recreating human thymus development in a dish using iPS cells
A team of researchers led by Professor Yoko Hamazaki and Assistant Professor Yann Pretemer (Department of Life Science Frontiers) has developed an in vitro model that faithfully recapitulates human thymic epithelial cell ...
Aug 26, 2025
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Two thirds of reproductive-aged women have at least one modifiable risk factor for birth defects, study reveals
An analysis using data from the National Health and Nutrition Examination Survey (NHANES) among 5,374 women of reproductive age indicates that two thirds of women within this demographic have at least one modifiable risk ...
Aug 26, 2025
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Repurposing a cholesterol drug may benefit DiGeorge syndrome patients with neuropsychiatric disease
Researchers have found that mitochondrial dysfunction in the blood-brain barrier (BBB) may lead to neuropsychiatric disease in some patients with DiGeorge syndrome.
Aug 20, 2025
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New guidance released to improve safety and outcomes of patent ductus arteriosus closure in premature infants
The Society for Cardiovascular Angiography & Interventions (SCAI) has published a new position statement that provides best-practice guidance for the treatment of patent ductus arteriosus (PDA) using transcatheter occlusion ...
Aug 18, 2025
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