Potential Gene Therapy for Limb Girdle Muscular Dystrophy Type 2D Granted Orphan Drug Designation

Limb Girdle Muscular Dystrophy Type 2D (LGMD2D), also called alpha-sarcoglycanopathy, is a form of muscular dystrophy which is caused by a defected SGCA gene. It causes the body to lose function of the alpha-sarcoglycan protein. The disease results in progressive muscle loss.

LGMD2D affects around 3 million people worldwide and doesn’t discriminate in regards to race, ethnicity, or gender. There are currently no effective treatments for the condition and there is no cure.

However, Myonexus Therapeutics is working to develop a gene therapy which could deliver the alpha-sarcoglycan genes into the body. Their hope is that this treatment would restore the proteins expression permanently. This  type of therapy could be life-changing for patients if approved.

Orphan Drug Designation

Myonexus has just announced that their potential gene therapy, called MYO-102, has received Orphan Drug Designation by the Food and Drug Administration (FDA).

This is a positive step in the development of this potential new therapy. It also confirms that the FDA sees potential in MYO-102 to be effective for patients.

The company is now planning their first human clinical trial for the drug and they’re hopeful about its continued success. Ultimately, researchers believe that this drug could improve symptoms for patients and restore much of their ability.

This population of patients has a critical need for new treatment options. This designation has brought renewed hope to this patient community that a therapy may be on the way.

You can read more about this gene therapy development and MYO-102’s new Orphan Drug Designation here.


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