Health Care
Guiding Treatment for Myotonic Dystrophy
Myotonic dystrophy (DM) is the most common form of muscular dystrophy in adults. This is a complex disease, affecting not just the muscles, but nearly every other organ system in the body. The signature manifestation is myotonia, an inability to consciously relax the muscles, coupled with progressive muscle weakness.
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The Case for Newborn Screening
Newborn screening allows babies born with life-threatening diseases to be treated before they show any signs of disease, which can lead to improved outcomes and maybe even a life free of symptoms.As therapies for neuromuscular diseases progress through the clinical pipeline and get approved by the U.S. Food and Drug Administration (FDA), the importance of newborn screening is heightened. Currently, it is recommended that states screen for Pompe disease and spinal muscular atrophy (SMA), and a screening for Duchenne muscular dystrophy (DMD) could be on the horizon. But despite these advancements, some states face implementation challenges due to scientific, economic and operational considerations.
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How Drugs Are Developed
Have you ever wondered what has to happen for the scientific community and pharmaceutical industry to develop a new drug and get it on the market, where physicians can prescribe it to treat a neuromuscular disease? Estimates vary, but it’s safe to say that, on average, out of 10,000 experimental compounds, one drug may make it to pharmacy shelves, and it will take at least 10 years and more than $1 billion to get it there.
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The Team Approach
Brendan is a curious, bright-eyed 7-year-old with a fun-loving attitude and a buoyant smile. He was diagnosed with Duchenne muscular dystrophy (DMD) when he was 23 months old. His mother, Colleen Labbadia, describes his diagnosis as long and heartbreaking but adds that there has been a bright spot. “Brendan is seen at the MDA Care Center at Nemours Children’s Hospital in Orlando, and we absolutely love it,” she says. “They have been our silver lining.”
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Impacted by Friedreich’s Ataxia? Share Your Voice!
An upcoming Friedreich’s ataxia (FA) Patient-Focused Drug Development (PFDD) meeting with the U.S. Food and Drug Administration (FDA) is your opportunity to tell the FDA and drug developers about challenges and burdens you have experienced with FA, and share your thoughts about what is most important to you in evaluating potential new treatments for the disease.
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A Good Beginning: Newborn Screening
For the first time in MDA history, we are seeing the development of specific drugs and therapies for the diseases in our program — many of which are the result of decades of MDA research. With potential new therapies in or nearing the approval stage, we are able to help infants with these diseases in a way never before possible: through a public health program called newborn screening (NBS).
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ANN Releases Guideline for LGMD Diagnosis and Care
This month, the American Academy of Neurology (AAN) released an evidence-based guideline for the diagnosis and treatment of all forms of limb-girdle muscular dystrophy (LGMD) and some forms distal muscular dystrophy (DD) – a development that's expected to improve the quality of care in these disorders.
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The FDA Approval Process: Can We Have This Drug Now?
To people faced with life-threatening diseases, the U.S. Food and Drug Administration (FDA) can seem like an indifferent obstacle, keeping them from treatments that would otherwise be available. But the reality is much more layered and complex.Here, MDA answers some frequently asked questions about how the FDA works to shed light on this topic.
Read MoreMedical Perspective: Scoliosis Surgery
Scoliosis and other spinal curvatures are common in neuromuscular diseases and often require surgical correction. Fortunately, today's surgeries are safer and more effective than those of earlier decades. (See Scoliosis Surgery: Setting the Record Straight, January 1997.) But that doesn't mean they're routine or simple.
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Going to the Emergency Room: Tips for People with Neuromuscular Diseases
When a medical emergency strikes — and the patient is a person with a neuromuscular disease — it’s not just getting to the emergency room quickly that’s critical. It’s also critical to ensure the ER staff understands the patient’s special needs caused by muscle disease.“Most ER doctors will NOT understand these diseases,” states Gregory Carter, medical director of the MDA Regional Neuromuscular Center at Providence St. Peter Medical Center in Olympia, Wash., and co-director of the MDA/ALS Center at the University of Washington Medical Center in Seattle.
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