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Who is a candidate for gene therapy? 

Gene Therapy

Candidates for MLD gene therapy (Lenmeldy) are specifically children with infantile and juvenile forms of  MLD. These patients must meet strict clinical and genetic criteria.  According to the FDA-approved indications,candidates must fall into one of these three categories.

Pre-symptomatic late infantile (PSLI)

  • Pre-symptomatic late infantile (PSLI): children with an expected disease onset at or before 30 months of age who do not show neurological symptoms

Pre-symptomatic early juvenile (PSEJ)

  • Pre-symptomatic early juvenile (PSEJ): children with an expected onset between 30 months and 7 years who show no symptoms

Early symptomatic early juvenile (ESEJ)

  • Early symptomatic early juvenile (ESEJ): Children with onset between 30 months and 7 years who can still walk independently or only show minor physical findings like abnormal reflexes

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