Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53.

C, Brogna; G, Coratti; M, Pane; V, Ricotti; S, Messina; A, D'Amico; C, Bruno; G, Vita; A, Berardinelli; E, Mazzone; F, Magri; F, Ricci; T, Mongini; R, Battini; L, Bello; E, Pegoraro; G, Baranello; SC, Previtali; L, Politano; GP, Comi; VA, Sansone; A, Donati; E, Bertini; F, Muntoni; N, Goemans; E, Mercuri

doi:10.1371/journal.pone.0218683

Public

Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53.

Shared by NobleBlocks on Jun 25, 2019 • 12:00 AM UTC

Authors:

Brogna C

Coratti G

Pane M

Abstract

Introduction The aim of this international collaborative effort was to report 36-month longitudinal changes using the 6MWT in ambulant patients affected by Duchenne muscular dystrophy amenable to skip exons 44, 45, 51 or 53.Materials and methods Of the 92 patients included in the study, 24 had delet...

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