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February 2005Research News -DMD Gene Therapy Trial Passes First Test -New Orphan Drug Aimed at Duchenne MD -Enzyme Found Safe, Well Tolerated in ALS More MDA News Pass it along! |
RESEARCH
NEWS
DMD Gene Therapy Trial Passes First Test
The trial, which MDA scientists hope to begin later this year, will test the safety of injecting a corrected gene for the muscle protein dystrophin tucked inside a modified adeno-associated virus. Read here for more about this important step toward MDA’s DMD gene therapy trial. New Orphan Drug Aimed at
Duchenne MD
PTC124, a compound that may be beneficial for about 15 percent of boys with Duchenne muscular dystrophy (DMD), has received Orphan Drug designation from the U.S. Food and Drug Administration (FDA). The designation, granted for potential therapies for rare diseases, provides pharmaceutical companies with tax advantages and other financial incentives for development of such therapies. PTC124 was developed by PTC Therapeutics of South Plainfield, N.J., in collaboration with MDA research grantees. Researchers believe PTC124 can target the mutations that lead to
the absence of the vital protein dystrophin in boys with DMD.
Enzyme Found Safe, Well Tolerated in
ALS
A pilot study of coenzyme Q10 in 31 people with ALS (Lou Gehrig’s disease) has shown the dietary supplement to be safe and well tolerated. MDA researchers will further study coenzyme Q10, which they think
may slow the progression of ALS.
MORE MDA NEWS
Still Time to Buy Shamrocks
Read here to learn how you or your business can participate in Shamrocks Against Dystrophy. Celebrating 50 Years of MDA Summer Camp
MDA summer camp counselors, who serve as companions to campers, must be at least 16 years old and able to lift a young person. Counselors help their campers with daily living activities and recreation. To learn about becoming a summer camp
volunteer or sponsor, contact your local MDA office, or call MDA
national headquarters at (800) 572-1717. Houston Couple Returns to Co-Chair ALS Division
Christopher and Reda Rice of Houston will serve a second year as co-chairpersons of MDA’s ALS Division. They’ll continue to help promote awareness of MDA’s leading efforts in the fight against ALS, or Lou Gehrig’s disease, through public appearances, media interviews, speaking engagements and public service announcements. Gilman Again Leads MDA Youth Programs
MDA National Youth Chairman Billy Gilman is continuing in the post this year. Gilman, who
first accepted the voluntary position in 2003, spearheads the
efforts of thousands of teens and young adults who help advance the
Association’s lifesaving mission. He supports MDA through
appearances at sponsor and volunteer events and through media
interviews. Gilman also performs and hosts youth segments on the
Jerry Lewis MDA Telethon.
Relive the Magic of the 2004 Telethon
You’ll see highlights of the broadcast’s tribute to late MDA
National Goodwill Ambassador Mattie J.T. Stepanek and comments from
his mom, Jeni; and performances by Elton John, Celine Dion, and
other favorites. The video includes portions of an interview with 2005 MDA
National Goodwill Ambassador Morgan Fritz and her parents, and
ends with Telethon star Jerry Lewis’ signature farewell song,
“You’ll Never Walk Alone.” Grade A
Click here to visit
the American Institute of Philanthropy’s Web site and receive a copy
of the report.
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