The results suggest that arsa-cel's disease-modifying effect in patients with early-onset MLD -- especially in children treated before symptoms developed -- is durable, Aiuti and colleagues said.

Since Rare Disease Day last year, several rare diseases have gained their first-ever FDA-approved drug, signifying progress in closing treatment gaps for these conditions.. 1. Niemann-Pick Disease ...

Khloe Garcia, 6, was recently diagnosed with Metachromatic leukodystrophy (MLD), a rare genetic disease that will leave her unable to walk, talk, see, or feed herself.

The approval makes atidarsagene autotemcel (arsa-cel [Lenmeldy]; Orchard Therapeutics) the first approved treatment for metachromatic leukodystrophy, a disease caused by a mutation in the ARSA ...

The study has introduced a new adeno-associated virus named PHP. eB (AAVPHP.eB) vector that carries the ARSA enzyme. This vector has shown promising results in treating the disease in a mouse model.

ARSA says it is fighting on behalf of the million Rohingya living in virtual detention in the western coastal state of Rakhine, who have few if any rights according to Myanmar’s government.

MLD is an inherited disease that leads to a deficiency of a key enzyme called arylsulfatase A (ARSA). Without enough of this enzyme, fatty substances build up in cells, causing damage to the ...

In a statement released on Thursday, ARSA said it had “no links with al-Qaeda, the Islamic State of Iraq and the Levant (ISIS), Lashkar-e-Taiba or any other transnational terrorist group”.

Adverse reactions because of myeloablative conditioning or atidarsagene autotemcel include febrile neutropenia, stomatitis, mucosal inflammation, veno-occlusive disease and anti-ARSA antibodies. For ...

ARSA says it is fighting on behalf of the million Rohingya living in virtual detention in the western coastal state of Rakhine, who have few if any rights according to Myanmar’s government.