96 results about "Muscle dystrophy" patented technology

Aspects of the disclosure relate to complexes and other aspects relate to formulations (e.g., aqueous, lyophilized forms) comprising such complexes (e.g., wherein each complex is of the exemplary formula shown below) comprising a phosphorodiamidate morpholino oligomer (e.g., useful for targeting DMD) covalently linked to an antibody (e.g., anti-TfR1 antibody). In some embodiments, the complexes are formulated with histidine (e.g., L-histidine) and sucrose at a specified pH (e.g., about 5.0 to 7.0). Also provided are uses of these formulations for treating a subject having a mutated DMD allele associated with Duchenne Muscular Dystrophy.

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.

The present disclosure relates to an oligonucleotide modulator for preventing or treating dystrophin deficiency-associated disorders (DDD), including Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). The oligonucleotide modulator comprises a sense nucleic acid strand and an antisense nucleic acid strand, the sense nucleic acid strand and the antisense nucleic acid strand being independently oligonucleotide strands of 16 to 35 nucleotides in length, and one nucleotide strand has at least 75% base homology or complementarity to a target selected from the promoter region of the target gene UTRN.

Methods for identifying compounds in the treatment of muscular dystrophies, include the use of disease relevant cells derived from a patient. Compounds identified by these methods are useful in the treatment of muscular dystrophy.

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 are compositions and methods of treating muscular dystrophies, including Duchenne Muscular Dystrophy (DMD) through administration of fibroblasts and modified fibroblasts systemically and locally. In certain embodiments, fibroblast cells are utilized for replacement of dystrophin through fusion and / or other means of horizontal gene transfer. In other embodiments, the disclosure teaches the use of fibroblasts for reduction of inflammatory reactions and / or immunological reactions which propagate and enhance myodestructive aspects of Duchenne Muscular Dystrophy. In other embodiments, fibroblasts are utilized as vectors for gene therapy and / or gene modifications approaches.

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.

Methods, systems, and techniques for controlling posture by using eye gaze technology to facilitate position changes in seating, such as in a wheelchair or bed, are provided. Example embodiments provide an Eye Gaze Posture Control System (“EGPCS”) which enables people with advanced motor neuron diseases or disabilities like amyotrophic lateral sclerosis or muscular dystrophy to use eye gaze technologies to control their seating and bed positioning. In one embodiment, the example EGPCS comprises a posture and pressure advisor, logging and telemetry logic, a posture control application, device interface(s) to one or more other devices that control aspects of the powered wheelchair or bed, and eye gaze actuation control. These components cooperate to adjust position of a seat or a bed using eye gaze technology as opposed to by a virtual cursor or by manual control.

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

To provide methods for treating patients with Duchenne muscular dystrophy.SOLUTION: Disclosed is a method for treating Duchenne muscular dystrophy in a subject in need thereof, the method comprising contacting the DNA of a muscle cell of the patient to a first nuclease that cleaves a first recognition sequence and a second nuclease that cleaves a second recognition sequence, where the first recognition sequence is upstream of a first exon of the dystrophin gene, and the second recognition sequence is downstream of the first exon of the dystrophin gene, and at least one second exon is removed from the dystrophin gene of the cell.SELECTED DRAWING: None

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.

A method of treating a muscular dystrophinopathy, or a condition associated therewith, in a subject by administering to the subject a therapeutically effective amount of a composition comprising ALY688. The muscular dystrophinopathy can be Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD), among others.

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.