Health Care
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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Weight Loss from a Wheelchair: My Plan
Note: Always consult your doctor before undertaking a weight loss plan. I have always had problems with my weight. From the time I was 5, I’ve had round, chubby (some would say pinch-able) cheeks — and my cheeks weren’t my only round part.When I was in high school I started really battling with weight. I decided to diet my senior year and lost 12 pounds. I thought that was the toughest dieting I would ever do, and I thought I was done with it. Wrong!
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OPMD: Surgery to Help Move Food Past Weak Throat Muscles
Starting with the tongue and moving down the throat and into the esophagus are a series of muscles that constrict and push food from the mouth to the stomach. The tongue and throat muscles weaken severely in OPMD, leading to choking, inhaling food into the lungs (“aspiration”) and lung infections (pneumonia). Speaking also can be adversely affected by weakening tongue and throat muscles.
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