Methods for treatment of charcot-marie-tooth disease with follistatin polypeptides
AssigneeAcceleron Pharma Inc.
InventorsRavindra Kumar, Asya Grinberg
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Abstract
The disclosure provides, in part, follistatin polypeptides that are suitable for use in local administration and methods for use.
Claims27
A method of treating a patient having Charcot-Marie-Tooth disease, the method comprising administering an effective amount of a follistatin fusion protein by an intramuscular route of administration to a targeted muscle of a patient in need thereof, wherein the follistatin fusion protein comprises a first amino acid sequence and a second amino acid sequence, wherein the first amino acid sequence comprises an amino acid sequence that is at least 90% identical to SEQ ID NO:
- 15 or 16, wherein the first amino acid sequence ends at the amino acid corresponding to any one of amino acids 291-302 of SEQ ID NO:
- 4;
- wherein the second amino acid sequence comprises a constant domain of an immunoglobulin G (IgG);
- and wherein the follistatin fusion protein inhibits signaling by myostatin in a cell-based assay.
Dependencies are inferred from the claim text.