Non-hormonal steroid modulators of NF-κβ for treatment of disease
The present invention relates to compounds and methods which may be useful as treatments of diseases.
1. A method of treating or reducing the symptoms of muscular dystrophy by increasing percentage of ejection fraction and increasing percentage of fraction shortening in a patient, the method comprising the administration, to the patient in need thereof, of a therapeutically effective amount of a compound for treating the muscular dystrophy, the compound having the structural formula
or a salt thereof,
wherein the therapeutically effective amount is greater than or equal to 2 mg/kg/day.
2. The method of claim 1 , wherein the muscular dystrophy is chosen from Duchenne muscular dystrophy, Becker muscular dystrophy, limb girdle muscular dystrophy, congenital muscular dystrophy, facioscapulohumeral muscular dystrophy, myotonic muscular dystrophy, oculopharyngeal muscular dystrophy, distal muscular dystrophy, and Emery-Dreifuss muscular dystrophy.
3. The method of claim 2 , wherein the muscular dystrophy is Duchenne muscular dystrophy.
4. The method of claim 1 , wherein the symptoms are chosen from muscle wasting, muscle degeneration, muscle inflammation, decreased percentage of ejection fraction, and decreased percentage of fraction shortening.
5. The method of claim 1 , wherein the improved ejection fraction and fraction shortening are measured by echocardiography.
6. The method of claim 1 , wherein the compound is administered orally.
7. The method of claim 1 , wherein the administration has a duration of at least about eight weeks.
8. A method of treating or reducing the symptoms of muscular dystrophy comprising the oral administration, to a patient in need thereof, of a therapeutically effective amount of
or a salt thereof,
wherein the therapeutically effective amount is greater than or equal to 2 mg/kg/day; and
the patient, following the administration, having improved ejection fraction and fraction shortening as measured by echocardiography.
9. The method of claim 8 , wherein the muscular dystrophy is chosen from Duchenne muscular dystrophy, Becker muscular dystrophy, limb girdle muscular dystrophy, congenital muscular dystrophy, facioscapulohumeral muscular dystrophy, myotonic muscular dystrophy, oculopharyngeal muscular dystrophy, distal muscular dystrophy, and Emery-Dreifuss muscular dystrophy.
10. The method of claim 9 , wherein the muscular dystrophy is Duchenne muscular dystrophy.
11. The method of claim 8 , wherein the symptoms are chosen from muscle wasting, muscle degeneration, muscle inflammation, decreased percentage of ejection fraction, and decreased percentage of fraction shortening.
12. The method of claim 8 , wherein the compound is administered orally.
13. The method of claim 8 , wherein the administration has a duration of at least about eight weeks.