Drugs

A look at symptoms, diagnosis, treatments and current MDA-supported research into this complex autoimmune disorder

posted on June 15, 2015 - 1:01pm
In recognition of June being National Myasthenia Gravis Awareness Month, MDA is examining the disease from several perspectives. This article offers an overview of signs, symptoms, diagnosis and treatments for myasthenia gravis (MG), as well as information on drugs that people with MG should avoid.

"Lumizyme has stopped the progression of my disease," says one of first to receive the drug since the FDA granted commercial approval

posted on June 21, 2010 - 4:25pm
Update (Aug. 4, 2014): The U.S. Food and Drug Administration (FDA) has expanded the approval of Lumizyme so that it can now be used to treat Pompe disease patients of all ages, including children younger than age 8. See FDA Expands Approval of Drug to Treat Pompe Disease to Patients of All Ages; Removes Risk Mitigation Strategy Requirements, Aug. 1, 2014.

The Food and Drug Administration has OK'd  the first U.S. treatment specifically for late-onset Pompe disease

posted on May 25, 2010 - 12:34pm
The U.S. Food and Drug Administration (FDA) has approved U.S. sales of the enzyme-replacement drug Lumizyme, the first treatment in the U.S. specifically for late-onset Pompe disease.

A decision on approval for Lumizyme, a treatment for Pompe disease, is expected in June

posted on March 19, 2010 - 9:25am
Biotechnology company Genzyme, of Cambridge, Mass., expects the U.S. Food and Drug Administration (FDA) to rule by June 17, 2010, on Genzyme's application to market its laboratory-developed enzyme Lumizyme (alglucosidase alfa) in the United States. Lumizyme and its near-twin, Myozyme, replace the acid maltase enzyme deficient in people with Pompe disease (acid alpha-glucosidase deficiency or acid...
posted on December 31, 2009 - 1:20am
QUEST Vol. 17, No. 1
A comprehensive set of clinical care recommendations for the diagnosis and management of Duchenne muscular dystrophy (DMD) is now available to medical professionals and families. The guidelines pull together recommendations previously released by other groups, such as the American Academy of Neurology, and clarify some areas in which consensus has been lacking.

After a year of planning, new recommendations for physicians caring for DMD patients have been released

posted on December 3, 2009 - 4:23pm
A comprehensive set of clinical care recommendations for the diagnosis and management of Duchenne muscular dystrophy (DMD) is now available to medical professionals and families. The complete recommendations,  in printable PDF format, can be read here:

A new National Institutes of Health questionnaire seeks public input to improve the agency’s information services

posted on October 10, 2009 - 10:55am
Important questions for Americans: What health care issues are important to you? Where do you get most of your health care information? If a loved one were sick with cancer or Alzheimer’s disease, where would you go for information? The National Institutes of Health (NIH) wants to know. A new NIH program seeks to obtain information from a wide cross-section of America that the agency hopes will...

FDA launches 'Access to Investigational Drugs’ Web site to aid patients and doctors

posted on August 20, 2009 - 2:00pm
The U.S. Food and Drug Administration (FDA) announced Aug. 12, 2009, that it has launched a Web site to help doctors and patients apply to obtain medications that have not yet been approved by the FDA ("investigational" drugs).