MDA Awards $6.6M to 25 Scientists Working in DMD, Other Neuromuscular Diseases

MDA Awards $6.6M to 25 Scientists Working in DMD, Other Neuromuscular Diseases

Twenty-five research grants totaling more than $6.6 million have been awarded by the Muscular Dystrophy Association  (MDA) to support studies into the mechanisms of neuromuscular diseases, including Duchenne and other muscular dystrophies.

The funding will allow researchers not only to investigate the causes of neuromuscular diseases, but also to develop new therapies — focusing especially on gene therapies — and improve the design of clinical trials.

Gene therapies involve either replacing, silencing, or removing a defective (mutated) gene, or they involve introducing new genes that confer other ways of working or offer protection.

“MDA’s latest group of awards aims to support the most promising research in a variety of neuromuscular diseases,” Lianna Orlando, the interim head of research at the MDA, said in a press release.

“Each funding award will address a key challenge or gap in knowledge and build upon the advances that have taken place in the last several years,” Orlando added.

In total, 25 research projects were funded. Five are led by young scientists, part of MDA’s commitment to supporting researchers in early stages of their careers.

Five awarded projects focus on Duchenne muscular dystrophy (DMD) — four in the U.S. and one at Monash University in Australia.

The Muscular Atrophy News forums are a place to connect with other patients, share tips and talk about the latest research. Check them out today!

One, led by Courtney Young, PhD, the CEO of MyoGene Bio in Los Angeles, will focus on assessing immune responses to single and repeated administration of an adeno-associated virus (a harmless virus); AAVS, as these viruses are called, are often used as transport vehicles to deliver gene therapies in animal models of DMD.

Young’s research team developed a DMD mouse model, which it then used to show that its gene-editing CRISPR/Cas9 platform can effectively increase the production of dystrophin, whose deficiency is the underlying cause of DMD, in human cells and in these mice.

“We are developing this therapeutic in MyoGene Bio in order to get it to patients as soon as possible,” Young said in an announcement posted on the MDA website. “Assessing the immune response will allow for safety to be assessed and strategies to re-dose the therapy to be developed, which would greatly increase the efficacy and applicability of the therapy.”

The project was awarded a three-year grant worth $299,592.

Additional diseases covered in the funded research include congenital muscular dystrophies (CMDs) or those present at birth, amyotrophic lateral sclerosis (ALS), Charcot-Marie-Tooth disease (CMT), facioscapulohumeral muscular dystrophy (FSHD), Friedreich’s ataxia (FA), inclusion body myositis (IBM), limb-girdle muscular dystrophy (LGMD), mitochondrial myopathy, myotonic dystrophy (DM), spinal-bulbar muscular atrophy (SBMA), and spinal muscular atrophy (SMA).

More information on these awards is available here.

Patricia holds a Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She has also served as a PhD student research assistant at the Department of Microbiology & Immunology, Columbia University, New York.
Total Posts: 307
Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Técnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.
×
Patricia holds a Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She has also served as a PhD student research assistant at the Department of Microbiology & Immunology, Columbia University, New York.
Latest Posts
  • cell therapy
  • gene therapy trial update
  • ACE-083
  • BB-301 gene therapy tests
Average Rating
0 out of 5 stars. 0 votes.
My Rating:

One comment

  1. Ram Chandra Paudel says:

    I am LGMD patient from Nepal,aged 50. Very difficult to stand up. I can stand by the help of wall touching or catching the window grill aside.

    Plz suggest me how I can get relief.

Leave a Comment

Your email address will not be published. Required fields are marked *