Duchenne Muscular Dystrophy News and Research

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Duchenne muscular dystrophy (DMD) is a progressive muscle disorder that causes the loss of both muscle function and independence. DMD is perhaps the most prevalent of the muscular dystrophies and is the most common lethal genetic disorder diagnosed during childhood today. Each year, approximately 20,000 children worldwide are born with DMD (one of every 3,500 male children).

Microtubule stiffening worsens skeletal muscle degeneration in Duchenne muscular dystrophy

University of Maryland (UM) researchers and collaborators report in the journal Science Signaling that skeletal muscle degeneration in Duchenne muscular dystrophy (DMD) is worsened by stiffening of the microtubule cytoskeleton that provides structure inside muscle cells.

23 Aug 2012

Laminin-111 protein therapy could treat Duchenne muscular dystrophy

A novel treatment in development at the University of Nevada School of Medicine for the most common form of muscular dystrophy is advancing towards human trials with a $308,000 boost from the Muscular Dystrophy Association.

23 Aug 2012

Antisense therapy shows promise in treating toxoplasmosis

A targeted approach to treating toxoplasmosis, a parasitic disease, shows early promise in test-tube and animal studies, where it prevented the parasites from making selected proteins. When tested in newly infected mice, it reduced the number of viable parasites by more than 90 percent, researchers from the University of Chicago Medicine report in the Proceedings of the National Academy of Sciences.

14 Aug 2012

Venom protein could treat Duchenne muscular dystrophy

While Spider-Man is capturing the imagination of theatergoers, real-life spider men in Upstate New York are working intently to save a young boy's life. It all began in 2009, when Jeff Harvey, a stockbroker from the Buffalo suburbs, discovered that his grandson, JB, had Duchenne muscular dystrophy. The disease is fatal. It strikes only boys, causing their muscles to waste away.

17 Jul 2012

Read-through molecule improves muscle function in DMD

A compound that results in a read-through on nonsense mutations in messenger RNA restores dystrophin expression in an animal model of Duchenne muscular dystrophy, research shows.

9 Jul 2012

Novel gene therapy approach may treat dysferlinopathies

The challenge of treating patients with genetic disorders in which a single mutated gene is simply too large to be replaced using traditional gene therapy techniques may soon be a thing of the past. A Nationwide Children's Hospital study describes a new gene therapy approach capable of delivering full-length versions of large genes and improving skeletal muscle function. The strategy may hold new hope for treating dysferlinopathies and other muscular dystrophies.

9 Jul 2012

New compound could treat certain types of genetic disorders in muscles

Scientists at UCLA have identified a new compound that could treat certain types of genetic disorders in muscles. It is a big first step in what they hope will lead to human clinical trials for Duchenne muscular dystrophy.

28 Jun 2012

First clinical trial to test cardiac drug regimen for DMD

The first landmark randomized clinical trial for a cardiac drug regimen in Duchenne muscular dystrophy (DMD) is testing whether earlier treatment can stop or slow down heart damage that usually kills people with the disease.

27 Jun 2012

PPMD to host first Transforming Duchenne Care Meeting from June 27-28

Parent Project Muscular Dystrophy (PPMD) will host the first Transforming Duchenne Care Meeting, June 27-28, 2012, immediately preceding the Annual Connect Conference in Fort Lauderdale, Florida.

26 Jun 2012

PPMD, AVI to participate in Rock 'n' Roll Seattle Marathon & Half Marathon

AVI BioPharma is teaming up with Parent Project Muscular Dystrophy (PPMD)'s endurance program, Run For Our Sons, to participate in the Rock 'n' Roll Seattle Marathon & Half Marathon on June 23, 2012. The goal is to raise money and awareness to help end Duchenne muscular dystrophy (Duchenne), the most common form of muscular dystrophy.

21 Jun 2012

Decision Resources forecasts the launch of four new agents to treat DMD by 2021

Decision Resources, one of the world's leading research and advisory firms focusing on pharmaceutical and healthcare issues, finds that interviewed experts unanimously identify a disease-modifying therapy as the highest unmet need in the treatment of Duchenne muscular dystrophy (DMD).

8 Jun 2012

Study identifies how skeletal muscle stem cells respond to muscle injury

A study conducted by Children's Hospital & Research Center Oakland scientists identifies how skeletal muscle stem cells respond to muscle injury and may be stimulated to improve muscle repair in Duchenne Muscular Dystrophy, a severe inherited disease of muscle that causes weakness, disability and, ultimately, heart and respiratory failure.

15 May 2012

Myomatrix 2012 conference to take place from April 22-24

The nation's leading scientists and clinicians exploring treatment breakthroughs for congenital muscular dystrophy will convene April 22-24 on the campus of the University of Nevada, Reno, where research by Dean Burkin has led to a potential therapy.

19 Apr 2012

KLF15 gene governs the body's ability to burn fat during exercise

While exercise is accepted universally as the most beneficial prescription physicians can write for patients, little is known about the molecular mechanisms that generate its widespread health benefits. Researchers from Case Western Reserve School of Medicine have shed light on this mystery by discovering that a genetic factor, Kruppel-like Factor 15 (KLF15), governs the body's ability to burn fat during exercise.

10 Apr 2012

TACT believes that Halo's HT-100 may effectively treat DMD

Halo Therapeutics, LLC, a clinical-stage biopharmaceutical company developing novel therapeutics for rare fibrotic diseases, announced today that the TREAT-NMD Advisory Committee on Therapeutics (TACT) has reviewed HT-100, Halo's drug candidate for Duchenne muscular dystrophy (DMD), and believes the compound has potential based on the proposed mechanism.

21 Mar 2012

New three-step process for DMD newborn screening

Investigators at Nationwide Children's Hospital, working with the DNA Sequencing Core Facility at the University of Utah, have developed an approach to newborn screening (NBS) for the life-threatening genetic disorder, Duchenne muscular dystrophy (DMD) and potentially other muscular dystrophies.

20 Mar 2012

Researchers discover molecular mechanism responsible for low back pain

Italian researchers at the Catholic University of Sacred Heart in Rome found an important molecular mechanism responsible for low back pain and other acute vertebral problems like cervical axial pain, all due to aging and degeneration of the vertebral column.

14 Mar 2012

Study examines abdomyosarcoma growth in mouse models of muscular dystrophy

Inflammation, cell division and cell differentiation that occur during skeletal muscle regeneration may provide an ideal environment for the highly malignant tumor, rhabdomyosarcoma to arise. These are the findings from a Nationwide Children's Hospital study that examined rhabdomyosarcoma growth in mouse models of muscular dystrophy.

23 Feb 2012

Prosensa secures €23M in new equity financing

Prosensa, the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet needs, announced today that it has raised €23 million in new equity financing.

25 Jan 2012

BioTime to market muscle progenitor cell lines bearing hereditary diseases

BioTime, Inc. today announced that it has elected to market progenitors of muscle stem cells bearing hereditary diseases. BioTime will produce the products from five human embryonic stem (hES) cell lines from Reproductive Genetics Institute (RGI) of Chicago, Illinois.

4 Jan 2012