Harlow is defying the odds after receiving groundbreaking gene therapy for a rare brain disorder. Her progress has been called "a miracle" by her family ...

THE WOODLANDS, Texas (KTRK) -- A family in The Woodlands is seeking help after two of their children were diagnosed with a deadly medical disorder. "It's so hard on their little bodies," said mother ...

Share on Facebook. Opens in a new tab or window Share on Bluesky. Opens in a new tab or window Share on X. Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window The FDA approved ...

A new medical treatment that costs more than $4 million for metachromatic leukodystrophy (MLD) was approved recently by the FDA. MLD was recognized as a disease in the early 1900s, although specifics ...

Credit: Shutterstock. OTL-200 is an ex vivo autologous hematopoietic stem cell gene therapy. The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application ...

CINCINNATI (WKRC) - There is promising new research for a genetic condition, which impacts thousands of families worldwide. A small company in the U.K. is giving one mother and son reason to hope. The ...

Five patients with adult-onset metachromatic leukodystrophy (MLD) underwent allo-SCT. Conditioning was reduced in intensity and grafts were obtained from voluntary unrelated donors. All but one graft ...

Currently, there are no approved treatments for MLD, a lysosomal storage disease caused by a deficiency in arylsulfatase A. The following article features coverage from the 17th Annual WORLDSymposium ...

TUBB4a leukodystrophy is an "incurable genetic condition" that affects one in 1,663 births, according to the Cleveland Clinic. First identified in 2014, it makes up a small percentage of the 50 rare ...

BUFFALO, N.Y. — Rep. Brian Higgins visited the New York State Center of Excellence in Bioinformatics and Life Sciences on Monday to announce more than $2 million in National Institutes of Health ...