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Methods for treatment of muscular dystrophies

A technology for muscular dystrophy and malnutrition, which is applied in the fields of medical formula, muscular system diseases, neuromuscular system diseases, etc., and can solve problems such as side effects

Inactive Publication Date: 2016-09-28
厄恩斯特.D.布什 +9
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

However, treatment with anabolic drugs can also be associated with serious side effects, including osteoporosis, high blood pressure, Cushing's syndrome, weight gain, cataracts, short stature, gastrointestinal symptoms, behavioral changes, and liver damage

Method used

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  • Methods for treatment of muscular dystrophies
  • Methods for treatment of muscular dystrophies
  • Methods for treatment of muscular dystrophies

Examples

Experimental program
Comparison scheme
Effect test

Embodiment 1

[0229] Example 1. Multidisciplinary Evaluation of Compound (I) Compared to Nandrolone and α-Methylprednisolone (PDN) in mdx of Exercise Effects of in vivo treatment on a mouse model of muscular dystrophy

[0230] introduce

[0231] The aim of this study was to test compound (I), a selective androgen receptor modulator (SARM) with muscle-specific effects, on chronic exercise in the mdx mouse model by a multidisciplinary approach of in vivo and ex vivo methods Impact. get duchener muscular dystrophy After the patients agreed to clinical use of glucocorticoids, the effect on compound (I) (30mk / kg, subcutaneous injection 6 days / week) was similar to that of those treated with α-methylprednisolone (PDN) (1mg / kg intraperitoneal injection 6 days / week) and compared with those of the anabolic nandrolone (5 mg / kg, subcutaneously 6 days / week).

[0232] Experiments describe results from ex vivo assays of key functional and morphological endpoints, corrected for methodological approac...

Embodiment 2

[0282] Example 2. Comparison of mdx mice treated with compound (I), nandrolone and α-methylprednisolone

[0283] Compound (I), nandrolone and α-methylprednisolone were administered 6 days per week to wild-type (Wt) and mdx mice. Figure 24 In vivo parameters of wild-type (Wt) and treated mdx mice treated with corn oil (Mdx+V1) or treated with compounds containing (I) (Mdx+compound (I)), or containing 5mg / kg nandrolone (Mdx+NAND), or containing water (Mdx+V2) or containing 1mg / kg α-methylprednisolone (Mdx+PDN) Composition treatment at 30 mg / kg. In each panel, vertical bars represent the mean ± S.E.M. of 5 to 7 animals. Significant differences between groups were assessed by correction for multiple comparisons using the ANOVA test and post-hoc Bonferroni t-test.

[0284] In (A), the vertical bar shows the body weight value (body weight) in g. No significant differences in values ​​for mdx mice (treated or untreated) were observed using the ANOVA test. In (B), vertical bar...

Embodiment 3

[0285] Example 3. Treatment of mdx mice with different amounts of compound (I)

[0286] Compound (I) was administered to wild-type (Wt) and mdx mice 6 days a week. Figure 25 In vivo parameters of wild-type (Wt) and treated mdx mice treated with corn oil (Mdx+ V1) Treatment or treatment with Compound (I) (Mdx+Compound (I)) in amounts of 0.3 mg / kg, 3 mg / kg and 30 mg / kg. In each panel, values ​​represent the mean±S.E.M. of 5 to 8 animals. Significant differences between groups were assessed by correction for multiple comparisons using the ANOVA test and post-hoc Bonferroni t-test.

[0287] In (A), the vertical bar shows the body weight value (body weight) in g. ANOVA test did not observe significant differences in BW at time 0, time 4 and time 6. Significant differences in BW were found at time 8 (F>3.9; p3.8; p最大 T0), week 4 (F 最大 T4), week 8 (F 最大 T8), week 12 (F 最大 T12), the vertical bar shows the maximum forelimb strength (forelimb force) in kg. ANOVA test showed s...

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Abstract

The invention discloses methods for treatment of muscular dystrophies. The present invention relates to, inter alia, treatment of muscle dystrophy (e.g., Duchenne Muscular Dystrophy), for example, using a composition, e.g., a composition comprising Compound (I), or a pharmaceutically acceptable salt, prodrug or metabolite thereof.

Description

[0001] priority interest [0002] This application claims priority under 35 U.S.C. §119(e) to U.S. Provisional Patent Application U.S.S.N. 61 / 895,832, filed October 25, 2013, which is incorporated herein by reference. Background technique [0003] Muscular dystrophies (MD) are a group of more than 30 genetic disorders characterized by progressive weakness and degeneration of the skeletal muscles that control movement. MD weakens the musculoskeletal system and hinders movement. MD is caused by progressive degeneration of skeletal muscle fibers. The disease is characterized by defects in muscle proteins and death of muscle cells and tissues. [0004] Dystrophinopathy is a group of muscular dystrophies caused by mutations in the dystrophin gene, which is located on the short arm of the X chromosome, in the Xp21 region [Kunkel et al. 1985; Monaco et al. 1985; Ray et al. 1985]. Of these, Duchenne muscular dystrophy (DMD), the most common dystrophinopathy, is caused by complete ...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): A61K31/395
CPCA61K31/4166A61P3/02A61P9/00A61P11/00A61P19/08A61P21/00A61P21/04A61P25/00A61P29/00A61K9/0053
Inventor 厄恩斯特.D.布什弗朗索瓦.尼克凯瑟琳.贾格尔施米特弗洛伦斯.S.纳穆罗兰.布兰克让-迈克尔.勒弗朗索瓦克里斯托夫.佩肖托皮埃尔.德普雷尼古拉斯.特里巴利奥皮特.T.B.P.威格林克
Owner 厄恩斯特.D.布什
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