MAP THE SMA: a Machine-learning Based Algorithm to Predict THErapeutic Response in Spinal Muscular Atrophy

RecruitingOBSERVATIONAL

Enrollment

247

Participants

Timeline

Start Date

April 1, 2023

Primary Completion Date

November 1, 2025

Study Completion Date

April 1, 2026

Conditions

Spinal Muscular Atrophy

Interventions

DRUG

disease modifying treatments

Patients will be enrolled if exposed to nusinersen, risdiplam, onasemnogene abeparvovec

Trial Locations (1)

RECRUITING

Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Roma

All Listed Sponsors

lead

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

OTHER