Learn About Gene Therapy for Muscular Dystrophy

ASGCT Staff - October 24, 2019

Muscular dystrophy is a group of genetic diseases that cause progressive muscle weakness and loss of muscle mass. There are a wide variety of muscular dystrophies, each caused by different gene mutations.

The American Society of Gene & Cell Therapy (ASGCT) released its latest resource profiling disease-specific treatment efforts, this time focusing on developing gene therapies for muscular dystrophy.

The resources on muscular dystrophy are now available on ASGCT.org. Muscular dystrophy is a group of genetic diseases that cause progressive muscle weakness and loss of muscle mass. There are a wide variety of muscular dystrophies, each caused by different gene mutations. For certain diseases, including limb-girdle muscular dystrophy (LGMD) and Duchenne muscular dystrophy (DMD), gene therapy might offer hope for slowing and managing symptoms

Watch ASGCT's Gene Therapy for DMD Video

Watch ASGCT's Gene Therapy for LGMD Video

The information on muscular dystrophy is part of the launch of a second round of material in ASGCT’s Patient Education program. ASGCT will release brand new resources weekly through the end of November on the Patient Education portal. Designed by ASGCT committee volunteers in coordination with patient advocacy groups, the patient-centered portal aims to educate and inform patients, families, and the public on the status and promise of gene and cell therapies.

The full schedule for ASGCT’s new Patient Education resources to be released during the remainder of 2019, excepting one-week breaks for the ASGCT Policy Summit and Thanksgiving, is as follows:

  • MPS (Mucopolysaccharidosis)—October 29
  • Hemophilia— November 12
  • Pompe—November 19
  • CAR T Basics—December 3

Let us know what you think of this second batch of resources. Send feedback via the ASGCT Contact form or at info@asgct.org

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