Metachromatic leukodystrophy, late infantile form Advocacy Hub | UniteRare

At a Glance

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Regulatory Watchboard

1 event

Mar 2024

Lenmeldy: FDA approved

treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ) or early symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD)

FDAcompleted

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

FINANCIAL LANDSCAPE SUMMARY

Total programs

No financial assistance programs currently listed for Metachromatic leukodystrophy, late infantile form.
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Approved Treatments

1 FDA-approved

Lenmeldy

(ATIDARSAGENE AUTOTEMCEL)Orphan drug

Orchard Therapeutics (Europe) Ltd

12.1 Mechanism of Action LENMELDY inserts one or more functional copies of the human ARSA complementary deoxyribonucleic acid (cDNA) into the patients...

Approved Mar 2024FDA label ↗

View full treatment details on disease page →