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 ...

This story is part of a series on the current progression in Regenerative Medicine. In 1999, I defined regenerative medicine as the collection of interventions that restore tissues and organs damaged ...

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 ...

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 ...

Please provide your email address to receive an email when new articles are posted on . Metachromatic leukodystrophy is an incurable rare genetic disease. At 5 years of age, 71% of children given ...

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.

Jaxtien Miller gets help peeling a piece of fruit from his mother Mercedes Boden at his home in Payson, Ill., after a physical therapy session on Nov. 4, 2019. Jax has a rare disease called ...

Metachromatic leukodystrophy (MLD) is a rare inborn error of metabolism leading to severe neurological symptoms and early death. Hematopoietic SCT (HSCT) is ...

Stable Levels of Engraftment and Reconstitution of Enzyme Activity in Patients with Up to 12 Months of Follow-up Indicate Similar Profiles Between Cryopreserved and Fresh Formulations MLD is a ...

If administered early, gene therapy has the potential to change the medical history of children born with metachromatic leukodystrophy (MLD), a rare and lethal neurodegenerative disease of genetic ...

NORTHRIDGE, LOS ANGELES (KABC) -- Amelia and Ella have a special bond. When Ella met Amelia in kindergarten, the fact that she was different never mattered to her. Ella just wanted to help her friend.