Metachromatic leukodystrophy is a rare genetic disorder that mainly affects young children and results in severe neurological symptoms accompanied by a loss of motor and intellectual capacities.

In an era when groundbreaking medicines are being developed every day, a new drug has emerged as the world’s most expensive. The drug is a gene therapy for an ultra-rare disease, and it costs millions ...

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 ...

It’s a new gene therapy for a rare, inherited disorder called metachromatic leukodystrophy (MLD) that occurs in 1 of every ...

About Lenmeldy Lenmeldy™ (atidarsagene autotemcel), formerly known as OTL-200, is the only approved therapy in the U.S. for the treatment of children with pre-symptomatic late infantile (PSLI), ...

The chief medical officer of a drug pricing watchdog is warning against the FDA granting full approval to drugs that were previously granted accelerated approval if they then go on to fail ...

Orchard Therapeutics has revealed its US pricing plans for Lenmeldy, its gene therapy for metachromatic leukodystrophy (MLD), placing a $4.25 million price tag on the one- The FDA has cleared its ...

The medications and treatment procedures have been patented. "Metachromatic leukodystrophy is found in 1 of every 50-70 thousand newborns," says Lab Head, Director of the Center for Precision and ...

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 ...

A family in Carlow have two children with metachromatic leukodystrophy (MLD) in need of a bone marrow transplant. Report shows Pat and Josie Brennan exercising their son Tommy. Josie Brennan ...