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 ...

Metachromatic leukodystrophy is a rare genetic disorder that affects between 1 and 9 people out of 100,000 and manifests itself mainly in childhood and adolescence—with 10 to 20% of cases observed in ...

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 ...

The Food and Drug Administration on Monday approved a gene therapy for metachromatic leukodystrophy, a devastating genetic disorder that eats away at affected children’s neurons. The medicine, ...

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 ...

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 ...

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 ...

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.

Cerebral adrenoleukodystrophy is a severe form of X-linked adrenoleukodystrophy characterized by white-matter disease, loss of neurologic function, and early death. Elivaldogene autotemcel (eli-cel) ...

Several rare disease patient populations received their first-ever FDA-approved drug since Rare Disease Day last year, signifying progress in closing treatment gaps for rare disease. Since Rare ...