June 2004


In This Issue:

Research News
 - Biotech Firm To Develop Gene Therapy For Duchenne
 - Creatine Helps Boys With DMD
 - New Gene Found In Charcot-Marie-Tooth Disease
 - MDA Researcher Elected To National Academy of Sciences

More MDA News

 - MDA and IAFF Celebrate 50 Years
 - Summertime Means Of Course, MDA Summer Camp
 - Mattie Is In Our Hearts
 - MDA's ALS Publication Gets Bright New Look & New Name
 

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Previous Issues:
May 2004

December 2003
October 2003
August 2003
July 2003
May 2003

March 2003


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Welcome to the MDA® e-update, the Muscular Dystrophy Association's online newsletter that reports MDA's research breakthroughs and other information to friends whose support helps to make our programs possible.


RESEARCH NEWS

BIOTECH FIRM TO DEVELOP
GENE THERAPY FOR DUCHENNE

MDA has awarded $1.6 million to the North Carolina biotechnology company Asklepios to develop gene therapy for Duchenne muscular dystrophy.

The company will develop and test a virus-based system designed to deliver a miniaturized dystrophin gene to the muscles of boys with DMD (very few girls are affected). Dystrophin is the muscle protein that's missing in this disease.

Extensive testing for safety will be required before the procedure can be tested in youngsters.

CREATINE HELPS BOYS WITH DMD

MDA-funded researchers saw small increases in strength in boys with Duchenne MD who took daily doses of the dietary supplement creatine during a recent, 4-month study.

Grip strength in the dominant hand and fat-free body mass (made mostly of muscle) increased in the participants, 30 boys who averaged 10 years in age.

Researchers want to investigate whether extended use of creatine might also improve breathing function, activities of daily living, and performance of tasks such as climbing stairs.

NEW GENE FOUND IN CHARCOT-MARIE-TOOTH DISEASE

Researchers have found a second gene that, when flawed, leads to the type 2A form of Charcot-Marie-Tooth disease.

A multinational research group studied seven families with this form of CMT, which causes weakness and atrophy of muscles in the hands and lower legs, foot deformities and some loss of sensation in the feet.

The MDA press release tells more, and includes a link to the study results published online in the journal Nature Genetics.

MDA RESEARCHER ELECTED TO NATIONAL ACADEMY OF SCIENCES

Mattie Stepanek

Kevin Campbell, a longtime MDA grantee at the University of Iowa in Iowa City, was elected in April to the National Academy of Sciences. The designation is one of the nation's highest honors for scientists.

Campbell's work has led to the identification of the molecular and genetic bases of several forms of muscular dystrophy, and provided a clearer understanding of the muscular dystrophy disease processes.

Campbell serves as a member of MDA's Scientific Advisory Committee.

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MDA NEWS

MDA AND IAFF CELEBRATE 50 YEARS

It's been five decades since MDA teamed up with the International Association of Fire Fighters, and the
partnership continues to thrive.

The IAFF is MDA's largest sponsor, having contributed more than $200 million to the fight against neuromuscular diseases, including $18.3 million last year alone.

The IAFF and MDA are celebrating the golden anniversary in many ways this year, including a special tribute to fire fighters planned for the 2004 Jerry Lewis MDA Telethon on Labor Day weekend.

Read about the rich history of this friendship, and some of the many ways that fire fighters across the country make a difference to the people served by MDA.

SUMMERTIME MEANS, OF COURSE, MDA SUMMER CAMP!

Mattie Stepanek

Thousands of youngsters nationwide eagerly await the arrival of summer so they can spend an incomparable week at an MDA summer camp.

It's been called "the best week of the year" and "better than Christmas" by kids with neuromuscular diseases. Each year some 4,000 youngsters enjoy a wide range of activities especially designed for people who have limited mobility or use wheelchairs. Swimming, boating, baseball, football and horseback riding, arts and crafts and talent shows are just part of the camp routine.

MATTIE IS IN OUR HEARTS

Mattie Stepanek

Mattie Stepanek, MDA's national goodwill ambassador for three years, has been through a severe ordeal that's kept him in intensive care in a Washington hospital since early March.

Recent updates on his condition are promising, but we continue to send our prayers and best wishes to 13-year-old Mattie and his mom, Jeni, and the outstanding team of medical professionals overseeing his care.

MDA'S ALS PUBLICATION GETS BRIGHT NEW LOOK & NEW NAME


Mattie Stepanek

MDA clients with amyotrophic lateral sclerosis are used to receiving valuable information and news in The MDA/ALS Newsletter.

The monthly publication has undergone a makeover, adding color and a more appealing layout, and with it a new name: the MDA/ALS Newsmagazine.

You can read the Newsmagazine's coverage of vital ALS information online. If you have ALS, you should be receiving the Newsmagazine at home; if you aren't, please notify your local MDA office.

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