ACE-083 1000 mcg muscular dystrophy research

90.00$

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Description

ACE-083 1000 mcg lyophilized – custom made per order – ships from the USA

Pharmacokinetics 

ACE-083 is a locally acting follistatin-based therapeutic that binds myostatin and other muscle regulators and has been shown to increase muscle mass and force in neuromuscular disease mouse models. This first-in-human study examined these effects. Methods: In this phase 1, randomized, double-blind, placebo-controlled, dose-ranging study in healthy postmenopausal women, ACE-083 (50-200 mg) or placebo was administered unilaterally into rectus femoris (RF) or tibialis anterior (TA) muscles as 1 or 2 doses 3 weeks apart. Results: Fifty-eight postmenopausal women were enrolled; 42 ACE-083 and 16 placebo. No serious adverse events (AEs), dose-limiting toxicities, or discontinuations due to AEs occurred. Maximum mean (± standard deviation [SD]) increases in RF and TA muscle volume were 14.5±4.5% and 8.9±4.7%, respectively. No significant changes in mean muscle strength were observed. Discussion: ACE-083 was well tolerated and resulted in significant targeted muscle growth. ACE-083 may have the potential to increase muscle mass in a wide range of neuromuscular disorders.

How does ACE-083 work?

FSHD is a disease characterized by the progressive weakening of muscles, starting in the face, shoulders, and upper arms.

ACE-083 is designed to increase the strength and function of specific muscles. The therapy contains a small molecule that binds to and inhibits select proteins in the TGF-beta protein superfamily, namely activins and myostatin, which reduce muscle growth.

Normally, muscle size is controlled by a balance of building and breaking down muscle. Exercise helps muscles to grow. If a person stops exercising, the muscles gradually reduce in size, due to the function of activins and myostatin, among other factors.

Inhibiting the TGF-beta family — sometimes called the “myostatin +” approach — reduces or slows muscle breakdown. This approach is thought to increase muscle mass and strength where the treatment is administered. Untreated muscles or other organs are not affected, which reduces the potential of side effects.

Sources:

[1] https://strongly.mda.org/fda-grants-orphan-drug-designation-to-acceleron-pharmas-ace-083-muscle-growth-drug-for-charcot-marie-tooth-disease/

[2] https://www.nmd-journal.com/article/S0960-8966(15)00304-1/abstract

[3] http://acceleronpharma.com/wp-content/uploads/2017/03/20141009-ACE-083-20141009-ACE-083-a-ligand-trap-for-members-of-the-TGFB-superfamily-increases-muscle-mass-loc.pdf

[4] https://www.ncbi.nlm.nih.gov/pubmed/29486514

[5] https://charcot-marie-toothnews.com/2018/07/26/preliminary-cmt-phase-2-trial-results-ace-083-presented/

FOR RESEARCH PURPOSES – NOT FOR HUMAN CONSUMPTION – image is for illustration only, all peptides ship in unlabeled vials with color coded caps for identification.

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