medical management

Pompe Disease, DMD: Newborn Screening Proposed

Proposals exploring the feasibility and advisability of implementating newborn screening for two disorders in MDA's program — Duchenne muscular dystrophy and Pompe disease— were presented to a federal advisory committee in a "virtual" meeting Jan. 31 and Feb. 1, 2013.

An ALS Biomarker? Brain Imaging Technique Pinpoints ALS, Disability

Advances in technology have led to greater sensitivity in imaging techniques, increasing scientists' ability to see into the brain and spinal cord. Now, a research team reports that a technique called magnetic resonance spectroscopic imaging (MRSI) can distinguish people with amyotrophic lateral sclerosis (ALS)  from people without the disease.

Low Body Fat Linked with Risk of Developing ALS

Anecdotal evidence has long described a positive association between being lean or underweight and developing amyotrophic lateral sclerosis (ALS). Now, results from a large-scale study conducted in Europe appear to confirm these observations, showing that individuals with more body fat have a decreased risk of developing the disease.

DMD: Which Steroid Regimen Is Best?

Prednisone and other drugs in the corticosteroid family have become standard treatment for Duchenne muscular dystrophy (DMD) in much of the world for almost a decade. In this disorder, they slow the decline of muscle function and prolong walking ability. However, corticosteroids have significant side effects, such as weight gain, bone loss, cataracts and behavior changes.

Adult-Onset MMD1/MMD2 and Juvenile-Onset MMD1

Medical Management

This section addresses medical management of the many symptoms of adult-onset MMD1 and MMD2, as well as juvenile-onset MMD1. These three forms of MMD share similar medical management strategies.

The subjects covered are:

MDA Webinar Discusses Spinal Bracing and Surgery in Children

A one-hour, MDA-sponsored webinar features two physicians and the parent of a child with spinal muscular atrophy (SMA) who has undergone bracing and surgery for a spinal curvature, as well as questions and answers from listeners.

Ventricular Assist Device Implanted in Man with Duchenne MD

A left ventricular assist device (LVAD), which helps the heart pump blood throughout the body, was implanted in the chest of a young man with Duchenne muscular dystrophy (DMD) at Cincinnati Children's Hospital Medical Center in September. The patient is 29-year-old Jason Williams, of Peebles, Ohio.

Patient Assistance Program Launched for Nuedexta

A new Patient Assistance Program can help people with a diagnosis of pseudobulbar affect (PBA) get a medication designed to treat the condition at low or no cost.

2012 AAN Meeting: Myasthenia Gravis Briefs

The 2012 annual meeting of the American Academy of Neurology, held in New Orleans April 21-28, included findings related to myasthenia gravis (MG).

2012 AAN Meeting: DMD, BMD Briefs

Update (June 1, 2012)­—  The brief Drugs help DMD-related cardiomyopathy has been updated to include a May 2012 podcast provided by Nationwide Children's Hospital's "This Month in Muscular Dystrophy."
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