P4.33 The Motor Function Measure (MFM), an outcome measure for neuromuscular disorders: Preliminary results in congenital muscular dystrophy (CMD)

2010 ◽  
Vol 20 (9-10) ◽  
pp. 668
Author(s):  
C.A.M. Payan ◽  
D. de Castro ◽  
S. Quijano-Roy ◽  
T. Stojkovic ◽  
C. Bérard ◽  
...  
Keyword(s):  
Muscular Dystrophy ◽  
Motor Function ◽  
Outcome Measure ◽  
Neuromuscular Disorders ◽  
Congenital Muscular Dystrophy ◽  
Preliminary Results ◽  
Function Measure

scholarly journals Promoting the use of Motor Function Measure (MFM) as outcome measure in patients with Duchenne Muscular Dystrophy (DMD) treated by corticosteroids

2015 ◽  
Vol 58 ◽  
pp. e139-e140
Author(s):  
A. Schreiber-Bontemps ◽  
S. Brochard ◽  
S. Fontaine-Carbonnel ◽  
S. Chabrier ◽  
V. Gautheron ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Motor Function ◽  
Outcome Measure ◽  
Function Measure

The gross motor function measure is valid for Fukuyama congenital muscular dystrophy

2017 ◽  
Vol 27 ◽  
pp. S226
Author(s):  
T. Sato ◽  
M. Adachi ◽  
K. Nakamura ◽  
M. Zushi ◽  
K. Goto ◽  
...  
Keyword(s):  
Muscular Dystrophy ◽  
Motor Function ◽  
Congenital Muscular Dystrophy ◽  
Gross Motor ◽  
Gross Motor Function ◽  
Gross Motor Function Measure ◽  
Function Measure

The gross motor function measure is valid for Fukuyama congenital muscular dystrophy

2017 ◽  
Vol 27 (1) ◽  
pp. 45-49 ◽  
Author(s):  
Takatoshi Sato ◽  
Michiru Adachi ◽  
Kaho Nakamura ◽  
Masaya Zushi ◽  
Keisuke Goto ◽  
...  
Keyword(s):  
Muscular Dystrophy ◽  
Motor Function ◽  
Congenital Muscular Dystrophy ◽  
Gross Motor ◽  
Gross Motor Function ◽  
Gross Motor Function Measure ◽  
Function Measure

A short form of gross motor function measure for Fukuyama congenital muscular dystrophy

2020 ◽  
Vol 42 (5) ◽  
pp. 383-388
Author(s):  
Takatoshi Sato ◽  
Michiru Adachi ◽  
Aya Matsuo ◽  
Masaya Zushi ◽  
Keisuke Goto ◽  
...  
Keyword(s):  
Muscular Dystrophy ◽  
Motor Function ◽  
Short Form ◽  
Congenital Muscular Dystrophy ◽  
Gross Motor ◽  
Gross Motor Function ◽  
Gross Motor Function Measure ◽  
Function Measure

The gross motor function measure™ is a valid and sensitive outcome measure for spinal muscular atrophy

2006 ◽  
Vol 16 (6) ◽  
pp. 374-380 ◽  
Author(s):  
Leslie Nelson ◽  
Hollis Owens ◽  
Linda S. Hynan ◽  
Susan T. Iannaccone ◽  
AmSMART Group
Keyword(s):  
Spinal Muscular Atrophy ◽  
Motor Function ◽  
Outcome Measure ◽  
Muscular Atrophy ◽  
Gross Motor ◽  
Gross Motor Function ◽  
Gross Motor Function Measure ◽  
Function Measure

Correlation between crural muscle fatty change and maximum motor function in Fukuyama congenital muscular dystrophy patients

2015 ◽  
Vol 25 ◽  
pp. S266-S267
Author(s):  
K. Ishigruo ◽  
K. Ishigaki ◽  
T. Sato ◽  
T. Murakami ◽  
M. Shichiji ◽  
...  
Keyword(s):  
Muscular Dystrophy ◽  
Motor Function ◽  
Congenital Muscular Dystrophy ◽  
Fatty Change

scholarly journals Tamoxifen in Duchenne muscular dystrophy (TAMDMD): study protocol for a multicenter, randomized, placebo-controlled, double-blind phase 3 trial

2019 ◽  
Author(s):  
Sara Nagy ◽  
Patricia Hafner ◽  
Simone Schmidt ◽  
Daniela Rubino-Nacht ◽  
Sabine Schädelin ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Motor Function ◽  
Open Label ◽  
Phase 3 ◽  
Double Blind ◽  
Safety And Efficacy ◽  
Open Label Extension ◽  
To Receive ◽  
Function Measure

Abstract BACKGROUND Duchenne muscular dystrophy (DMD) is an inherited neuromuscular disorder of childhood with a devastating disease course. Several targeted gene therapies and molecular approaches have been or are currently tested in clinical trials; however, a causative therapy is still not available and best supportive care is limited to oral glucocorticoids with numerous long-term side effects. Tamoxifen is a selective estrogen receptor regulator, and shows besides its antitumor activity also antioxidant actions and regulatory roles in the calcium homeostasis. In a mouse model of DMD, oral tamoxifen significantly improved muscle strength and reduced muscle fatigue. This multicenter, randomized, double-blind, placebo controlled phase 3 trial aims to demonstrate safety and efficacy of tamoxifen over placebo in pediatric patients with DMD. After completion of the double-blind phase, an open label extension of the study will be offered to all participants. METHODS/DESIGN At least 71 ambulant and up to 20 non-ambulant patients with DMD are planned to be enrolled at multiple European sites. Patients will be randomly assigned to receive either tamoxifen 20mg or placebo daily over 48 weeks. In the open-label extension phase, all patients will be offered to receive tamoxifen for further 48 weeks. The primary endpoint of the double-blind phase is defined as the change of the D1 domain of the motor function measure in ambulant patients or a change of the D2 domain in non-ambulant patients under tamoxifen compared to placebo. Secondary outcome measures include change in timed function tests, quantitative muscle testing, and quantitative MRI of thigh muscles. Laboratory analyses including biomarkers of tamoxifen metabolism and muscle dystrophy will also be assessed. DISCUSSION The aim of the study is to investigate whether tamoxifen can reduce disease progression in ambulant and non-ambulant DMD patients over 48 weeks. Motor function measure comprises the primary endpoint, whereas further clinical and radiological assessments and laboratory biomarkers are performed to provide more data on safety and efficacy. An adjacent open label extension phase is planned to test if earlier initiation of the treatment with tamoxifen (verum arm of double blind phase) compared to a delayed start can reduce disease progression more efficiently.

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