55 results about "Muscle dystrophy" patented technology

Substituted pyridazinone compounds, conjugates, and pharmaceutical compositions for use in the treatment of neuromuscular diseases, such as Duchenne Muscular Dystrophy (DMD), are disclosed herein. The disclosed compounds are useful, among other things, in the treating of DMD and modulating inflammatory inhibitors IL-1, IL-6 or TNF-α.

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

Substituted pyridazinone compounds, conjugates, and pharmaceutical compositions for use in the treatment of neuromuscular diseases, such as Duchenne Muscular Dystrophy (DMD), are disclosed herein. The disclosed compounds are useful, among other things, in the treating of DMD and modulating inflammatory inhibitors IL-1, IL-6 or TNF-α.

The present invention relates to peptides, in particular cell-penetrating peptides, and to conjugates of such cell-penetrating peptides with a therapeutic molecule. The present invention further relates to use of such peptides or conjugates in methods of treatment or as a medicament, especially in the treatment of genetic disorders and in particular muscular dystrophies such as Duchenne muscular dystrophy.

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 binding moiety (e.g., antibody)-oligonucleotide (e.g., PMO) conjugates and pharmaceutical compositions that induce an alteration in an incorrectly spliced dystrophin mRNA transcript to induce DMD exon 45 skipping. Also described herein include methods for treating muscle dystrophy including Duchenne muscular dystrophy that comprises administering such conjugates or a pharmaceutical composition that induces alteration in an incorrectly spliced dystrophin mRNA transcript to induce DMD exon 45 skipping.

New dosing regimens for treating muscular dystrophy in a patient suffering from Duchenne muscular dystrophy (DMD) with an antisense oligonucleotide conjugate that causes skipping of an exon in the human dystrophin gene are described. Also described is a method of treating a patient with an antisense oligomer CPP conjugate and a magnesium supplement.

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.

The present invention relates to modified recombinant agrin proteins or fragments thereof and recombinant chimeric laminin-nidogen proteins or fragments thereof, nucleic acid sequences encoding these proteins, vectors containing these nucleic acid sequences, compositions comprising these proteins, and the use of these proteins in the prevention and / or treatment of congenital muscular dystrophies, in particular (laminin-α2) LAMA2-associated muscular dystrophy.

Disclosed herein are binding moiety (e.g., antibody)- oligonucleotide (e.g., PMO) conjugates and pharmaceutical compositions that induce an alteration in an incorrectly spliced dystrophin mRNA transcript to induce DMD exon 45 skipping. Also described herein include methods for treating muscle dystrophy including Duchenne muscular dystrophy that comprises administering such conjugates or a pharmaceutical composition that induces alteration in an incorrectly spliced dystrophin mRNA transcript to induce DMD exon 45 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.

Novel antisense oligonucleotide conjugates that cause exon skipping in the human dystrophin gene and their use in methods of treating Duchenne muscular dystrophy (DMD) in patients suffering from the disease are described.

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 binding moiety (e.g., antibody)- oligonucleotide (e.g., PMO) conjugates and pharmaceutical compositions that induce an alteration in an incorrectly spliced dystrophin mRNA transcript to induce DMD exon 45 skipping. Also described herein include methods for treating muscle dystrophy including Duchenne muscular dystrophy that comprises administering such conjugates or a pharmaceutical composition that induces alteration in an incorrectly spliced dystrophin mRNA transcript to induce DMD exon 45 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 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 α7 integrin modulatory agents and methods of using such to treat muscular disorders, disease, degeneration, and / or muscular dystrophy. The methods include administering an effective amount of an α7 integrin modulatory agent to the subject with a muscular disorder, disease, degeneration, and / or dystrophy, wherein the α7 integrin modulatory agent increases α7 integrin expression or activity as compared to α7 integrin expression or activity prior to treatment, thereby treating the subject. Also disclosed are methods of enhancing muscle regeneration, repair, or maintenance in a subject and methods of enhancing α7 integrin expression by use of the disclosed α7 integrin modulatory agents. Methods of preventing or reducing muscle injury or damage in a subject are also disclosed.

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.

The present invention relates to a modified recombinant agrin protein or a fragment thereof and a recombinant chimeric laminin-nidogen protein or a fragment thereof, to nucleic acid sequences encoding these proteins, to vectors comprising these nucleic acid sequences, to compositions comprising these proteins and to uses of these proteins in the prophylaxis and / or treatment of congenital muscular dystrophies, in particular (laminin-α2) LAMA2-related muscular dystrophy.

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

Among other things, the present disclosure provides oligonucleotide compositions and methods thereof. In some embodiments, oligonucleotides comprise various chemical modifications of sugars, nucleobases, and / or intemucleotidic linkages and patterns thereof and are useful for exon skipping. In some embodiments, the present disclosure provides technologies useful for skipping exon 51 of dystrophin (DMD) transcripts. In some embodiments, the present disclosure provides technologies usefill for modulating DMD transcript splicing. In some embodiments, the present disclosure provides methods for preventing or treating conditions, disorders or diseases including Duchenne muscular dystrophy.

Described herein are methods of treating muscular dystrophy in a subject, comprising administration of a recombinant AAV vector AAVrh74.tMCK.SGCA using a systemic route of administration and at a dose of about 1.0×1012 vg / kg to about 5.0×1015 vg / kg. Further disclosed are methods of expressing alpha-sarcoglycan gene in a cell or in a subject in need thereof, decreasing a serum CK level, and increasing alpha-sarcoglycan positive fibers in muscle tissue of a subject.

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.

The disclosure provides methods useful for treating and / or preventing muscular dystrophies, such as Duchenne's muscular dystrophy (DMD), and / or other muscle wasting disorders by modulating prolyl hydroxylase 3 (PHD3).

To provide pyridazinone compounds, conjugates and pharmaceutical compositions for use in the treatment of neuromuscular diseases, such as Duchenne Muscular Dystrophy (DMD).SOLUTION: For example, the invention provides a substituted pyridazinone compound with the structure in the figure, or a salt thereof, and a pharmaceutical composition thereof.SELECTED DRAWING: None