“If you cannot measure it, you cannot improve it”. Outcome measures in Duchenne Muscular Dystrophy: current and future perspectives

被引：0

作者：

Silvia Benemei ^{[1
]}

Francesca Gatto ^{[1
]}

Luca Boni ^{[2
]}

Marika Pane ^{[3
]}

机构：

[1] Pfizer Italy,Medical Affairs

[2] U.O. Epidemiologia Clinica,Nemo Clinical Centre, Fondazione Policlinico Universitario A. Gemelli IRCSS

[3] IRCCS Ospedale Policlinico San Martino,undefined

[4] Università Cattolica del Sacro Cuore,undefined

来源：

Acta Neurologica Belgica | 2025年 / 125卷 / 1期

关键词：

Duchenne muscular dystrophy; Clinical outcomes; North star ambulatory assessment (NSAA); 6-Minute walking test (6MWT); 4 stairs climbed (4SC); Performance of the upper limb (PUL);

D O I：

10.1007/s13760-024-02600-2

中图分类号：

学科分类号：

摘要：

Duchenne Muscular Dystrophy (DMD) is an X-linked recessive neuromuscular disorder primarily affecting males, caused by mutations in the dystrophin gene. The absence of dystrophin protein leads to progressive skeletal muscle degeneration. Recent advances in the therapeutic landscape underscore the need to identify appropriate outcome measures to assess treatment efficacy in ambulant and non-ambulant DMD patients, across clinical and research settings. This is essential for accurately evaluating new treatments and attributing therapeutic benefits.

引用

页码：1 / 12

页数：11