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Learn about
LENMELDY

Actor portrayal. MLD is a recessive inherited disease.
Actor portrayal. MLD is a recessive inherited disease.

Learn about
LENMELDY

GENE THERAPY WITH LENMELDY

LENMELDY is a blood stem cell-based gene therapy for the treatment of:

Pre-symptomatic late infantile (PSLI) MLD in infants under 30 months old who have not yet shown symptoms
Pre-symptomatic early juvenile (PSEJ) MLD in young children between 30 months and 7 years old who have not yet shown symptoms
Early symptomatic early juvenile (ESEJ) MLD in young children between 30 months and 7 years old with early-stage symptoms

LENMELDY uses the child’s own blood stem cells to correct early onset* MLD. The cells are first collected from the child in a specialized clinic, then the cells are sent to a gene therapy lab where they insert a working copy of the ARSA gene into the cells. Those cells can then be returned to the child in a specially trained hospital.

ELIGIBILITY ASSESSMENT

MLD specialists at your Qualified Treatment Center (QTC) will perform a series of tests to determine whether or not your child is eligible for treatment with LENMELDY.

Your child will receive treatment with LENMELDY at a QTC. These QTCs are highly qualified facilities that have been trained to administer LENMELDY. Your doctor will provide a referral to the QTC.

*See full indication.

IMPORTANT SAFETY INFORMATION

What is LENMELDY?

LENMELDY is a one-time gene therapy developed to treat children with pre-symptomatic late infantile, pre-symptomatic early juvenile and early symptomatic early juvenile, referred to as early-onset, metachromatic leukodystrophy (MLD). MLD is caused by a defect in the arylsulfatase A (ARSA) gene, which causes the body to produce reduced or no ARSA enzyme. LENMELDY is made specifically for each child, using the child’s own blood stem cells, and adding functional copies of the ARSA gene to their cells. This may allow you to produce sufficient ARSA enzyme to stop or slow the progression of MLD symptoms.

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