Myostatin or growth differentiation factor 8 (GDF8) is a naturally occurring protein in muscles. It belongs to a family of proteins called transforming growth factor-beta or TGFβ. Myostatin, produced through instructions from the MSTN gene, regulates muscle cell growth and differentiation. Specifically, it limits skeletal muscle growth to protect muscles from becoming excessively large.

Secreted by the muscle cells, myostatin production increases with age.

Myostatin inhibitors are a group of molecules that block myostatin, and may work to improve muscle mass and strength in children with muscle-wasting diseases. They are being investigated as potential treatments for diseases such as muscular dystrophy.

Myostatin inhibitors in development

Several myostatin inhibitors are being evaluated as potential treatments for different types of muscular dystrophy.

RG6206

RG6206 (also known as RO7239361 or BMS986089) is an investigational molecule to possibly treat Duchene muscular dystrophy (DMD). It was first developed by Bristol-Myers Squibb and is now licensed to Roche. This molecule works by binding to myostatin and limiting its function, and so encourage muscle growth.

ACE-083

ACE-083 is an investigational therapy being developed by Acceleron to possibly treat facioscapulohumeral muscular dystrophy (FSHD) and Charcot-Marie-Tooth disease (CMT). It is a small molecule designed to bind to and block myostatin and another TGFβ protein called activin, increasing the strength and function of muscles.

Domagrozumab (PF-06252616)

Domagrozumab (PF-06252616) was a candidate intended to treat DMD being developed by Pfizer. A monoclonal antibody against myostatin, it was designed to work by binding to myostatin and blocking its activity. Development of domagrozumab was discontinued in August 2018 after it failed to show effectiveness against placebo in a Phase 2 study of ambulatory boys with DMD.

 

Last updates 07/15/2019

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Muscular Dystrophy News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified healthcare providers with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

Vijaya Iyer is a freelance science writer for BioNews Services. She has contributed content to their several disease-specific websites, including cystic fibrosis, multiple sclerosis, muscular dystrophy, among others. She holds a PhD in Microbiology from Kansas State University, where her research focused on molecular biology, bacterial interactions, metabolism, and animal models to study bacterial infections. Following the completion of her PhD, Dr. Iyer went on to complete three postdoctoral fellowships at Kansas State University, University of Miami and Temple University. She joined BioNews Services to utilize her scientific background and writing skills to help patients and caregivers remain abreast with important scientific breakthroughs.
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Vijaya Iyer is a freelance science writer for BioNews Services. She has contributed content to their several disease-specific websites, including cystic fibrosis, multiple sclerosis, muscular dystrophy, among others. She holds a PhD in Microbiology from Kansas State University, where her research focused on molecular biology, bacterial interactions, metabolism, and animal models to study bacterial infections. Following the completion of her PhD, Dr. Iyer went on to complete three postdoctoral fellowships at Kansas State University, University of Miami and Temple University. She joined BioNews Services to utilize her scientific background and writing skills to help patients and caregivers remain abreast with important scientific breakthroughs.
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