Please provide your email address to receive an email when new articles are posted on . Biotech firm SynaptixBio has entered into a licensing agreement with the Children’s Hospital of Philadelphia, ...

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 ...

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 ...

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 ...

The US Food and Drug Administration (FDA) has approved the first gene therapy for metachromatic leukodystrophy (MLD), a rare and debilitating genetic disease affecting the brain and nervous system.

WASHINGTON, Aug. 16, 2021 /PRNewswire/ -- Cure MLD, an initiative of The Calliope Joy Foundation, and the National Organization for Rare Disorders, Inc. today launched a wide-reaching study to ...

Finding showed treatment with Lenmeldy significantly extended severe motor impairment-free survival in children with PSLI MLD compared with untreated natural history children. The Food and Drug ...

A genetic medicine at the heart of a $387 million Kyowa Kirin acquisition has won FDA approval, a regulatory decision that makes the one-time treatment the first approved therapy for the rare ...

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 ...

Gene therapy, if administered early, preserves motor and cognitive functions. Newborn screening is crucial but not yet widespread. The study is the result of 20 years of research at the San ...