Quest Magazine

Traditionally, outcome measures— the observations investigators make in a clinical trial to decide whether one treatment is better than another or better than a placebo — are determined by factors in a disease that are considered important by physicians and other professionals.

But in recent years, there has been increasing interest in finding out what matters most to people with the condition being studied.

MDA is conducting a survey designed to assess the experiences of parents in the United States whose babies underwent newborn screening at the time of birth, with an eye toward the future possibility of newborn screening tests being recommended for certain neuromuscular diseases in which therapy development is advancing rapidly.

An experimental treatment for type 1 myotonic muscular dystrophy (MMD1, or DM1) has corrected several aspects of the disease in an MMD1 mouse model.

A year after treatment with an experimental antisense oligonucleotide dubbed ASO 445236 ended, positive effects remained apparent in the mice.

If you're the parent of a young child who uses a power mobility device, researchers at the University of British Columbia (UBC) and Sunny Hill Health Centre for Children — both in western Canada — are interested in hearing from you. The study is open to U.S. residents.

New Jersey biopharmaceutical company PTC Therapeutics recently declared its intention to do whatever it takes — including an additional clinical trial — to seek approval for ataluren (formerly PTC124) as a treatment for Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) caused by non

Phrixus Pharmaceuticals in Ann Arbor, Mich., reported July 19, 2012, that its experimental drug Carmeseal demonstrated a beneficial effect on the diaphragm (the primary breathing muscle) in mice with a disease resembling Duchenne muscular dystrophy (DMD).