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7 Result(s)

Recruiting trial = Recruiting trial
; Ongoing trial = Ongoing trial
; Funded by an IRDiRC member = ; Member of a ERN =

National clinical trial(s)

ITALY

LAZIO
ROMA

A Phase 2b Efficacy and Safety Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne Muscular Dystrophy and Becker Muscular Dystrophy - IT
IRCCS Ospedale Pediatrico Bambino Ges¨ - SEDE SAN PAOLO
UnitÓ di Malattie Neuromuscolari e Neurodegenerative - Laboratorio di Medicina Molecolare

ITALY

LAZIO
ROMA

Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients - IT
IRCCS Ospedale Pediatrico Bambino Ges¨ - SEDE SAN PAOLO
UnitÓ di Malattie Neuromuscolari e Neurodegenerative - Laboratorio di Medicina Molecolare

ITALY

LAZIO
ROMA

Funded by an IRDiRC memberAn Open-Label Study for Previously Treated Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy (Phase III) - IT
IRCCS Ospedale Pediatrico Bambino Ges¨ - SEDE SAN PAOLO
UnitÓ di Malattie Neuromuscolari e Neurodegenerative - Laboratorio di Medicina Molecolare

ITALY

LAZIO
ROMA

Recruiting trial
Ongoing trial
Prospective open label trial to evaluate the maintenance of the efficacy and the long term safety of EPI-743 in children with Leigh Disease (Phase II) - IT
IRCCS Ospedale Pediatrico Bambino Ges¨ - SEDE SAN PAOLO
UnitÓ di Malattie Neuromuscolari e Neurodegenerative - Laboratorio di Medicina Molecolare

ITALY

LAZIO
ROMA

Recruiting trial
Ongoing trial
Open Label, Long-term Safety, Tolerability, and Efficacy Study of GIVINOSTAT in All DMD Patients Who Have Been Previously Treated in One of the GIVINOSTAT Studies - IT
IRCCS Ospedale Pediatrico Bambino Ges¨ - SEDE SAN PAOLO
UnitÓ di Malattie Neuromuscolari e Neurodegenerative - Laboratorio di Medicina Molecolare

Multinational clinical trial(s)