Scientists have identified novel genetic interactions that may contribute to congenital heart disease (CHD), a common birth defect.

Norrie disease is an extremely rare, inherited genetic disorder that causes vision loss and was first described in Denmark in ...

A toddler was successfully treated for a rare genetic disease, spinal muscular atrophy, after world-first in-womb therapy.

People who carry a certain variation of this repeated DNA sequence have more than double the risk (2.2 times higher)of ...

Scientists at the Icahn School of Medicine at Mount Sinai and collaborators have identified novel genetic interactions that may contribute to congenital heart disease (CHD), a common birth defect.

Qure's AMT-130 shows 80% Huntington's progress reduction, eyeing FDA approval. Click here to read why I rate QURE stock a Buy ...

A new discovery offers hope for Huntington’s disease. This discovery provides hope that a DNA repair process may help slow or ...

Medical genetics department expands services, designated as Centre of Excellence under National Policy for Rare Diseases.

Brandi Muhle was 9 years old when her 35-year-old mother, an Omaha police officer and a marathon runner, was first diagnosed ...

Researchers developed a new gene-editing strategy that dramatically boosts the effectiveness of gene therapies in the liver, a breakthrough that could lead to new treatments for about 700 genetic ...

Though learning that your child has a heart defect like a ventricular septal defect can be frightening, understanding the ...

Berlin, Germany, and Research Triangle Park, N.C., USA, Feb. 19, 2025 (GLOBE NEWSWIRE) -- Not intended for UK Media Regenerative Medicine Advanced Therapy (RMAT) designation follows Phase Ib 36-month ...