18 мар. 2024 г. · The US Food and Drug Administration approved Lenmeldy (atidarsagene autotemcel), the first FDA-approved gene therapy indicated for the treatment of children.

The optimal therapy for MLD would provide persistent and high level expression of ARSA in the CNS. Gene therapy using adeno-associated virus (AAV) is an ideal ...

19 мар. 2024 г. · The Food and Drug Administration on Monday approved the first and only gene therapy for the treatment of children with metachromatic leukodystrophy (MLD).

22 янв. 2022 г. · They represent the first demonstration of arrest or slowing of progression of both central and peripheral nervous system disease caused by MLD.

Metachromatic leukodystrophy (MLD) is a rare inherited lysosomal storage disease caused by deficiency of arylsulfatase A (ARSA), due to mutations in the ARSA ...

18 мар. 2024 г. · Lenmeldy is the first and only treatment in the U.S. for early onset MLD. Today the FDA approved Lenmeldy (atidarsagene autotemcel), a ...

20 мар. 2024 г. · A new gene therapy for the fatal genetic disorder metachromatic leukodystrophy, or MLD, will carry a wholesale price of $4.25 million, ...

Within the leukodystrophies, there are active gene therapy clinical trials for metachromatic leukodystrophy, adrenoleukodystrophy, globoid cell leukodystrophy, ...

In addition, it is not curative. The gene therapy approach uses a lentivirus to introduce a functioning ARSA gene into the patient's own blood stem cells, which ...

16 окт. 2020 г. · EMA has recommended granting a marketing authorisation in the European Union for the gene therapy Libmeldy to treat metachromatic leukodystrophy (MLD).