Premium
Clinical investigation in Duchenne muscular dystrophy: Penicillamine and vitamin E
Author(s) -
Fenichel Gerald M.,
Brooke Michael H.,
Griggs Robert C.,
Mendell Jerry R.,
Miller J. Phillip,
Moxley Richard T.,
Park Jane H.,
Provine Michael A.,
Florence Julaine,
Kaiser Kenneth K.,
King Wendy M.,
Pandya Shree,
Robison Jenny,
Signore Linda
Publication year - 1988
Publication title -
muscle and nerve
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.025
H-Index - 145
eISSN - 1097-4598
pISSN - 0148-639X
DOI - 10.1002/mus.880111110
Subject(s) - duchenne muscular dystrophy , penicillamine , medicine , placebo , muscular dystrophy , muscle contracture , clinical trial , vitamin , surgery , pathology , alternative medicine
A double‐blind controlled trial of penicillamine and vitamin E against placebo was conducted in 106 boys with Duchenne muscular dystrophy. Nine dropped out of the study after being randomized. Patients were evaluated using an established protocol that measures muscle strength, joint contractures, functional grade, and pulmonary function. The trial lasted for 18 months. The power of the study to detect a 75% slowing of the disease was 0.95 at a P < 0.05. No therapeutic effect was observed in the patients taking penicillamine and vitamin E as compared with those taking placebo.