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.

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

posted on August 17, 2009 - 5:00pm
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).

Insmed halts development of its drug Iplex for MMD1 and ALS, at least for now

posted on July 27, 2009 - 5:00pm
The Richmond, Va., biopharmaceutical company Insmed announced July 27, 2009, that it will not supply its experimental drug Iplex to any new patients with amyotrophic lateral sclerosis (ALS) for the foreseeable future, and that it intends to analyze the available data on Iplex for ALS and type 1 myotonic dystrophy (MMD1, or DM1) before deciding whether to proceed with development of the drug for...

Drug improves insulin sensitivity but not other aspects of MMD1

posted on June 25, 2009 - 5:00pm
The drug Iplex, developed by the Richmond, Va., biopharmaceutical company Insmed, did not improve muscle function, strength or endurance in a phase 2 trial in type 1 myotonic dystrophy (MMD1, or DM1), the company announced June 25, 2009. (See Insmed Announces Results.)

The approval of Myozyme in 2006, after decades of research, has transformed Pompe disease from a severely disabling or fatal condition into a treatable, chronic disorder. But responses to the drug vary, its cost is high, and some questions remain unanswered.

posted on January 1, 2009 - 2:25pm
QUEST Vol. 16, No. 1
Mia Hanley of Cranston, R.I., looks pretty much like any 4-year-old preschooler. She’s a normal height and weight, walks well and has good language skills. In fact, when she started preschool at age 3, her fine motor skills were so good that she didn’t qualify for special services in that area. Looking at Mia, most people would never suspect she spends every other Friday at Hasbro Children’s...

Specialists, support groups and stimulant medications help families cope with the unusual cognitive and personality effects of type 1 myotonic dystrophy

posted on September 1, 2008 - 2:27pm
QUEST Vol. 15, No. 5
*Many people interviewed for this article asked not to be identified to protect the privacy of affected family members. “Everybody knows the word apathy,” says a California woman whose 25-year-old daughter’s type 1 myotonic dystrophy (MMD1, sometimes called DM1) was diagnosed just a few years ago. “People use the word loosely. I don’t think it does justice to the reality of this disease.”
posted on July 1, 2008 - 3:01pm
QUEST Vol. 15, No. 4
Letters to the editor discuss overcoming obstacles, facing everyday difficulties, and sleepiness and the drug Provigil.

Clinical trials involve risks, rights, responsibilities

posted on May 1, 2008 - 3:23pm
QUEST Vol. 15, No. 3
Twenty-year-old Carlie Brinker of Millersburg, Ohio, knows what it’s like to have a chronic, disabling condition and to have to take medications that come with a panoply of side effects.