Target Information

Target General Infomation
Target ID
T87749
Former ID
TTDR00823
Target Name
Growth/differentiation factor 8
Gene Name
MSTN
Synonyms
GDF-8; Myostatin; MSTN
Target Type
Clinical Trial
Disease Disuse muscle atrophy; Cancercachexia [ICD9: 140-229, 799.4; ICD10: R64]
Duchenne muscular dystrophy [ICD10: G71.0]
Muscular atrophy [ICD9: 728.2; ICD10: M62.5]
Muscular dystrophy [ICD10: G71.0]
Muscle atrophy [ICD10: M62.5]
Function
Acts specifically as a negative regulator of skeletal muscle growth.
BioChemical Class
Growth factor
UniProt ID
O14793
Sequence
MQKLQLCVYIYLFMLIVAGPVDLNENSEQKENVEKEGLCNACTWRQNTKSSRIEAIKIQI
LSKLRLETAPNISKDVIRQLLPKAPPLRELIDQYDVQRDDSSDGSLEDDDYHATTETIIT
MPTESDFLMQVDGKPKCCFFKFSSKIQYNKVVKAQLWIYLRPVETPTTVFVQILRLIKPM
KDGTRYTGIRSLKLDMNPGTGIWQSIDVKTVLQNWLKQPESNLGIEIKALDENGHDLAVT
FPGPGEDGLNPFLEVKVTDTPKRSRRDFGLDCDEHSTESRCCRYPLTVDFEAFGWDWIIA
PKRYKANYCSGECEFVFLQKYPHTHLVHQANPRGSAGPCCTPTKMSPINMLYFNGKEQII
YGKIPAMVVDRCGCS
Drugs and Mode of Action
Drug(s) ACE-031 Drug Info Phase 2 Duchenne muscular dystrophy [522999]
AMG 745 Drug Info Phase 2 Muscular atrophy [522779]
LY2495655 Drug Info Phase 2 Disuse muscle atrophy; Cancercachexia [523751]
PF-06252616 Drug Info Phase 2 Muscular dystrophy [525019]
SAR391786 Drug Info Phase 2 Muscle atrophy [524475]
Stamulumab Drug Info Phase 1/2 Duchenne muscular dystrophy [521630]
Inhibitor ACE-031 Drug Info [532125]
Antagonist AMG 745 Drug Info [544190]
Modulator LY2495655 Drug Info [544346]
Target Expression Profile (TEP) and Drug Resistance Mutation (DRM)
TEP EXP Info
Pathways
PANTHER Pathway TGF-beta signaling pathway
WikiPathways Hypertrophy Model
References
Ref 521630ClinicalTrials.gov (NCT00104078) Study Evaluating MYO-029 in Adult Muscular Dystrophy. U.S. National Institutes of Health.
Ref 522779ClinicalTrials.gov (NCT00975104) AMG 745 in Subjects With Age-associated Muscle Loss. U.S. National Institutes of Health.
Ref 522999ClinicalTrials.gov (NCT01099761) Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy. U.S. National Institutes of Health.
Ref 523751ClinicalTrials.gov (NCT01505530) A Phase 2 Study of LY2495655 in Participants With Pancreatic Cancer. U.S. National Institutes of Health.
Ref 524475ClinicalTrials.gov (NCT01963598) Study of the Safety and Efficacy of REGN1033 (SAR391786) in Patients With Sarcopenia. U.S. National Institutes of Health.
Ref 525019ClinicalTrials.gov (NCT02310763) A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy. U.S. National Institutes of Health.
Ref 530761Myostatin (GDF-8) as a key factor linking muscle mass and bone structure. J Musculoskelet Neuronal Interact. 2010 Mar;10(1):56-63.
Ref 532125A single ascending-dose study of muscle regulator ACE-031 in healthy volunteers. Muscle Nerve. 2013 Mar;47(3):416-23.
Ref 544190The role of myostatin in muscle wasting: an overview. J Cachexia Sarcopenia Muscle. 2011 September; 2(3): 143-151.
Ref 544346Myostatin inhibitors as therapies for muscle wasting associated with cancer and other disorders. Curr Opin Support Palliat Care. 2013 November; 7(4): 352-360.

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