28 results about "Muscle dystrophy" patented technology

Disclosed herein are polynucleic acid molecules, pharmaceutical compositions, and methods for treating muscle dystrophy (DM1).

Disclosed herein are antibody oligonucleotide conjugates and pharmaceutical compositions that induce an alteration in an incorrectly spliced dystrophin mRNA transcript to induce exon 44 skipping. Also described herein include methods for treating muscle dystrophy including Duchenne muscular dystrophy that comprises administering antibody oligonucleotide conjugates or a pharmaceutical composition that induces alteration in an incorrectly spliced dystrophin mRNA transcript to induce exon 44 skipping.

Disclosed herein are antibody oligonucleotide conjugates and pharmaceutical compositions that induce an alteration in an incorrectly spliced dystrophin mRNA transcript to induce exon 44 skipping. Also described herein include methods for treating muscle dystrophy including Duchenne muscular dystrophy that comprises administering antibody oligonucleotide conjugates or a pharmaceutical composition that induces alteration in an incorrectly spliced dystrophin mRNA transcript to induce exon 44 skipping.

Disclosed herein are antibody-oligonucleotide conjugates and pharmaceutical compositions comprising the antibody-oligonucleotide conjugates that induce an alteration in an incorrectly spliced dystrophin mRNA transcript to induce DMD exon 52 skipping. Also described herein include methods for treating muscle dystrophy including Duchenne muscular dystrophy by administering antibody-oligonucleotide conjugates or a pharmaceutical composition comprising the antibody-oligonucleotide conjugates that induce an alteration in an incorrectly spliced dystrophin mRNA transcript to induce DMD exon 52 skipping.

Disclosed are methods of treating muscular atrophy diseases, such as spinal muscular dystrophy or muscular dystrophy, using matrix-bound vesicles (MBV). Compositions for the treatment of muscular atrophic diseases are also disclosed.

The present disclosure provides a method of treating muscle myopathy, including muscle dystrophies and cardiomyopathies, by administering stable, long-lasting vasoactive intestinal peptide therapeutic agents. These agents include one or more elastin-like peptides and can be administered at a low-dose.

Disclosed herein are antibody oligonucleotide conjugates and pharmaceutical compositions that induce an alteration in an incorrectly spliced dystrophin mRNA transcript to induce exon 44 skipping. Also described herein include methods for treating muscle dystrophy including Duchenne muscular dystrophy that comprises administering antibody oligonucleotide conjugates or a pharmaceutical composition that induces alteration in an incorrectly spliced dystrophin mRNA transcript to induce exon 44 skipping.

Disclosed herein are polynucleic acid molecules, pharmaceutical compositions, and methods for treating muscle dystrophy (DM1).

Disclosed herein are antibody-oligonucleotide conjugates and pharmaceutical compositions comprising the antibody-oligonucleotide conjugates that induce an alteration in an incorrectly spliced dystrophin mRNA transcript to induce DMD exon 50 skipping. Also described herein include methods for treating muscle dystrophy including Duchenne muscular dystrophy by administering antibody-oligonucleotide conjugates or a pharmaceutical composition comprising the antibody-oligonucleotide conjugates that induce an alteration in an incorrectly spliced dystrophin mRNA transcript to induce DMD exon 50 skipping.

Disclosed herein are polynucleic acid molecules, pharmaceutical compositions, and methods for treating muscle dystrophy (DM1).

Disclosed herein are antibody-oligonucleotide conjugates and pharmaceutical compositions comprising the antibody-oligonucleotide conjugates that induce an alteration in an incorrectly spliced dystrophin mRNA transcript to induce DMD exon 50 skipping. Also described herein include methods for treating muscle dystrophy including Duchenne muscular dystrophy by administering antibody-oligonucleotide conjugates or a pharmaceutical composition comprising the antibody-oligonucleotide conjugates that induce an alteration in an incorrectly spliced dystrophin mRNA transcript to induce DMD exon 50 skipping.

Disclosed herein are polynucleic acid molecules, pharmaceutical compositions, and methods for treating muscle dystrophy (DM1).

The present disclosure is directed to a method of treating muscular dystrophy in a subject in need thereof. In certain embodiments, the method comprises administering to the subject an AAV vector, such as an AAVrh74 vector, and an enzyme that cleaves IgG. A method of preparing a subject for gene therapy for muscular dystrophy is provided, the method comprising administering to the subject an enzyme that cleaves IgG prior to administering a recombinant AAVrh74 viral vector.

Some embodiments relate to recombinant adeno-associated virus (rAAV) delivery of polynucleotides for treating muscular dystrophy resulting from the duplication of DMD exon 2. The invention provides rAAV products and methods of using the rAAV in the treatment of muscular dystrophy

The invention provides a biomarker for detecting Duchenne muscular dystrophy and application of the biomarker, and belongs to the technical field of biological medicine. The biomarker is selected from one or more of the following genes: a COL1A2 gene, a COL3A1 gene, a COL6A6 gene, an MYH3 gene and a VCAN gene. The inventor performs comprehensive transcriptome analysis on normal muscle tissues and three kinds of dystrophy muscle tissues with different pathological degrees, and aims to identify key genes crossing the whole muscular dystrophy pathology and mark the severity of the key genes. Therefore, a basis is provided for exploring a potential treatment method for improving pathological conditions of muscular dystrophy. The AUC values of the COL1A2, COL3A1, COL6A6, MYH3 and VCAN genes in the diagnosis of the Duchenne muscular dystrophy reach 0.976 at most, and are obviously higher than the diagnosis values of the traditional indexes, so that the test efficiency is good.

Histone Deacetylase inhibitors having potential therapeutic utility for cancer, neuromuscular dystrophies and neurodegenerative disorders and cardiac dysfunctions are disclosed. The invention provides compounds, its preparation method and formulations which are useful for the treatment of these disorders.

Disclosed herein are antibody-oligonucleotide conjugates and pharmaceutical compositions comprising the antibody-oligonucleotide conjugates that induce an alteration in an incorrectly spliced dystrophin mRNA transcript to induce DMD exon 50 skipping. Also described herein include methods for treating muscle dystrophy including Duchenne muscular dystrophy by administering antibody-oligonucleotide conjugates or a pharmaceutical composition comprising the antibody-oligonucleotide conjugates that induce an alteration in an incorrectly spliced dystrophin mRNA transcript to induce DMD exon 50 skipping.

Disclosed herein are polynucleic acid molecules, pharmaceutical compositions, and methods for treating muscle dystrophy (DM1).

Disclosed herein are antibody-oligonucleotide conjugates and pharmaceutical compositions comprising the antibody-oligonucleotide conjugates that induce an alteration in an incorrectly spliced dystrophin mRNA transcript to induce DMD exon 52 skipping. Also described herein include methods for treating muscle dystrophy including Duchenne muscular dystrophy by administering antibody-oligonucleotide conjugates or a pharmaceutical composition comprising the antibody-oligonucleotide conjugates that induce an alteration in an incorrectly spliced dystrophin mRNA transcript to induce DMD exon 52 skipping.

Provided herein are anti-myostatin antibodies which bind myostatin. The anti-myostatin antibodies of the disclosure are useful for the treatment of muscle dystrophies by blocking myostatin signaling. Also provided herein are methods of use of the anti-myostatin antibodies.

The disclosure relates to methods and compositions comprising p38 kinase inhibitors and agents that modulate the expression of DUX4 and catarrhal genes including but not limited to ZSCAN4, LEUTX, PRAMEF2, TRIM43, MBD3L2, KHDC1L, RFPL2, CCNA1, SLC34A2, TPRX1, PRAMEF20, TR1M49, PRAMEF4, PRAME6, PRAMEF15, or ZNF280A. Methods useful for the treatment of a disease associated with abnormal expression of DUX4 and catarrhal genes (e.g., peritoneo-scaphobrachial muscular dystrophy) are disclosed.

Disclosed herein are antibody-oligonucleotide conjugates and pharmaceutical compositions comprising the antibody-oligonucleotide conjugates that induce an alteration in an incorrectly spliced dystrophin mRNA transcript to induce DMD exon 52 skipping. Also described herein include methods for treating muscle dystrophy including Duchenne muscular dystrophy by administering antibody-oligonucleotide conjugates or a pharmaceutical composition comprising the antibody-oligonucleotide conjugates that induce an alteration in an incorrectly spliced dystrophin mRNA transcript to induce DMD exon 52 skipping.

Disclosed herein are nucleic acids comprising novel and optimized nucleotide sequences encoding human follistatin. Further disclosed herein are compositions comprising such nucleic acids and methods of uses thereof for treating muscle-related diseases and conditions (e.g., sarcopenia or muscle dystrophy) in a subject. Adeno-associated virus (AAV) vectors carrying such nucleic acid (s) and uses thereof are further described.