Genetic modification is the only credible path to restoring the blight-wracked American chestnut. Bruce Beehler is a ...

D Molecular Therapeutics is undervalued, with its lead candidate 4D-150 showing potential. Click here to find out why I rate FDMT 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 ...

The high cost of sickle cell gene therapies is pushing private insurers and Medicaid programs to develop new payment models.

Solid said that the first patients to receive its Duchenne gene therapy all produced high levels of the target protein, an ...

AAV-Navβ1 gene therapy effectively reduces seizures and prolongs life in a mouse model of SCN1B-linked Dravet syndrome, ...

On this episode, an infant with a harrowing diagnosis defies all odds after receiving a $2 million genetic treatment. Then, a ...

A UK study found more than 80% of 80 SMA type 1 children on a DMT had scoliosis or other abnormal spinal curvature needing ...

Test your knowledge of spinal muscular atrophy (SMA) with this quiz covering its genetic causes, diagnosis, and treatment ...

Vincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma ...

By Balambal Suryanarayanan CRISPR-Cas9 is a repurposed gene editing tool that allows scientists to cut, replace, and insert pieces of DNA in precise regions along the strand. Courtesy of NIH Image ...

Discover Stoke Therapeutics' innovative RNA-based treatments like zorevunersen for Dravet syndrome, with a promising pipeline ...