METHODS OF TREATING FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY WITH LOSMAPIMOD
Provided herein, in part, are methods of treating facioscapulohumeral muscular dystrophy in a patient in need thereof, comprising administering losmapimod to the patient.
1 . A method of treating facioscapulohumeral muscular dystrophy (FSHD) in a patient in need thereof, comprising:
administering 15 mg, twice daily, of losmapimod to the patient for at least 40 weeks,
wherein after the at least 40 weeks the patient has improved muscle health as measured by whole-body musculoskeletal magnetic resonance imaging (WB-MSK-MRI).
2 . A method of reducing fat accumulation in a muscle at a high risk of progression of facioscapulohumeral muscular dystrophy (FSHD), in a patient in need thereof, comprising:
administering 15 mg, twice daily, of losmapimod for at least 40 weeks.
3 . The method of claim 1 or 2 , wherein after the at least 40 weeks the muscle has reduced fat accumulation as measured by whole-body musculoskeletal magnetic resonance imaging (WB-MSK-MRI).
4 . The method of claim 2 or 3 , comprising imaging the muscle using whole-body musculoskeletal magnetic resonance imaging (WB-MSK-MRI) to determine a correlation between a measure of muscle health and a clinical outcome assessment of the patient.
5 . The method of claim 4 , wherein the measure of muscle health is selected from the group consisting of muscle fat fraction (MFF), muscle fat infiltration (MFI), and lean muscle volume (LMV).
6 . The method of claim 4 , wherein the clinical outcome assessment is selected from the group consisting of a reachable workspace (RWS), a relative surface area (RSA), and an assessment made from a Timed Up and Go (TUG) test.
7 . The method of any one of claims 2 - 7 , wherein the muscle, prior to administering losmapimod to the patient, is characterized by a muscle fat infiltration (MFI) of less than about 0.10 and a muscle fat fraction (MFF) of less than about 0.50.
8 . The method of any one of claims 2-7 , wherein the muscle, prior to administering losmapimod to the patient, is characterized by a muscle fat infiltration (MFI) of greater than or equal to about 0.10 and a muscle fat fraction (MFF) of less than about 0.50.
9 . The method of any one of claims 1-8 , comprising administering 15 mg, twice daily, of losmapimod to the patient for at least 48 weeks.
10 . The method of any one of claims 1-9 , wherein the FSHD is FSHD1.
11 . The method of any one of claims 1-10 , wherein the muscle is selected from the group consisting of a muscle of an upper extremity and a muscle of a lower extremity.
12 . The method of any one of claims 1-10 , wherein the muscle is selected from the group consisting of shoulder abductor and an ankle dorsiflexor.
13 . The method of claim 12 , wherein the shoulder abductor is selected from the group consisting of a bilateral shoulder abductor, a dominant shoulder abductor, and a non-dominant shoulder abductor.