61 results about "Muscular dystrophy" patented technology

Disclosed herein are improved methods for treating muscular dystrophy, such as DMD, BMD, or FSHD. Various combination therapies and monotherapies are provided.

The invention discloses application of hydrogen-rich water in preparation of a drug for treating Duchenne type muscular dystrophy, and particularly relates to the technical field of medicines.The random control experiment is adopted, homologous c57 mice serve as a normal control group, mdx mice are randomly divided into a model group and a hydrogen-rich water group, and the exercise ability is evaluated through a four-limb holding power test and a rotating bar test; detecting mouse creatine kinase by an enzyme-linked immunosorbent assay; carrying out hematoxylin-eosin, NADH-tetrazole reductase and improved Goori three-color dyeing, and carrying out cytochrome C oxidase, succinate dehydrogenase and SDH-COX combined dyeing to observe the pathological change of the skeletal muscle tissue; the invention discloses that the muscle injury condition of a Duchenne type muscular dystrophy model mouse is improved by the hydrogen-rich water through a mitochondrial protection mechanism, and the hydrogen-rich water has a relatively high application prospect and a relatively high economic value.

The present invention provides an oral dosage form for, and a therapeutically effective dose for the management and / or treatment of muscular dystrophy using Sitagliptin or its pharmaceutically acceptable salt, alone or in combination with other therapeutically effective agents.

The present application provides an engineered circular RNA capable of recruiting adenosine deaminase acting on RNA (ADAR; arRNA) and its application in treating Duchenne muscular dystrophy (DMD).

The group of inventions relates to the field of medicine, pharmacology and the chemical and pharmaceutical industry and includes an improved method for producing (10S, 13S, 16R, 17R)-17-hydroxy-10,13,16-trimethyl-17-propanoyl-7,8,12,14,15,16-hexahydro-6H-cyclopenta[a]phenanthrene-3-one (compound of formula (I)) and the use thereof for industrial batch, semi-continuous and continuous chemical production. Also described is a method for producing an intermediate compound, Vamorolone Acetate. The method for producing the compound of formula (I) is carried out according to the schema depicted. The production method makes it possible to produce the compound of formula (I) with a purity suitable for the use thereof in a finished dosage form for subjects requiring treatment for Duchenne muscular dystrophy. Thus, the technical results of the present invention consist in an improved production method which results in: a high yield and chemical purity, including stereoisomeric purity, of the compound of formula (I) and intermediate products in the synthesis of the compound of formula (I), which are suitable for use in a finished dosage form for subjects requiring treatment for Duchenne muscular dystrophy; the adaptability of the synthesis of the compound of formula (I) to industrial production; environmentally-friendly production; and a decrease in the formation of undesirable by-products.

Disclosed are conjugates of an oligonucleotide and a peptide covalently bonded or linked to the oligonucleotide via a linker that target a human dystrophin gene, compositions including the same, and methods of use thereof.

Described herein in various embodiments are compositions comprising (a) a cyclic peptide; and (b) an antisense compound, wherein the antisense compound targets exon 44 of the DMD gene in a pre-mRNA sequence.

The present disclosure provides compositions related to binding of various sarcoglycan proteins, which are relevant for their role in numerous genetic disorders, including limb girdle muscular dystrophy. The disclosure includes proteins, antibodies and / or fragments thereof and associated polynucleotide constructs. The disclosure further provides methods for manufacturing said compositions and other uses for the same.

The present disclosure is concerned with piperidinedione compounds, pharmaceutical compositions comprising the compounds, and methods of treating disorders associated with the presence of a premature termination codon such as, for example, cystic fibrosis, Duchenne muscular dystrophy, aniridia, Becker muscular dystrophy, spinal muscular atrophy, Hurler syndrome, hemophilia, epidermolysis bullosa (e.g., dystrophic (DEB) form, junctional (JEB) form). Usher syndrome, and cancer, using the compounds. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present invention.

The present disclosure relates generally to alkyne containing pharmaceutical agents, and in particular, to phenylethynyl-thiophene based compounds. More particularly, the present disclosure provides a class of compounds that can inhibit and / or attenuate apoptosis via caspase 3 for the treatment of various degenerative disorders. Additionally, the present disclosure relates to methods for treating specific degenerative disorders such as amyotrophic lateral sclerosis (ALS), Huntington's disease, epilepsy, spinal cord injury, complication due to diabetes, multiple sclerosis (MS), muscular dystrophy (MD), Parkinson's disease (PD), irritable bowel syndrome (IBS) and Alzheimer's disease (AD) in a patient comprising administering to the patient an effective amount of a present compound.

The present invention provides novel irisin peptides (e.g, irisin glycosylation mutants and biologically active fragments thereof). Also provided are methods for preventing or reducing degeneration of dopaminergic neurons and / or preventing or ameliorating at least one motor deficit in a subject in need thereof, such as in a subject with α-synucleinopathy, using the novel irisin peptides to modulate irisin-induced integrin signaling. The novel irisin peptides can also be used in methods for increasing expression of brain-derived neurotrophic factor (BDNF), and / or treating or preventing neurological diseases or disorders that would benefit from decreased neuronal cell death and / or increased neuronal survival in a subject. In addition, the novel irisin peptides may be used in methods for preventing or treating muscular atrophy or muscular dystrophy.

Aspects of the present disclosure relate to conjugates comprising a muscle targeting agent covalently linked to a molecular payload. In some embodiments, the muscle targeting agent specifically binds to an internalized cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits the expression or activity of DUX4. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or an RNAi oligonucleotide.

The present invention provides novel fluorinated cyclic derivatives of sulfonamides and sulfones, compositions thereof, and methods for preparing and using them, which are useful for treating various diseases and disorders related to TRPML activity, such as neurodegenerative diseases, lysosomal storage disorders, muscular dystrophy, common neurodegenerative diseases associated with aging, diseases associated with oxidative stress or reactive oxygen species (ROS), and aging.

Disclosed herein are compounds for treating and / or managing muscular dystrophy. According to the embodiment of the invention, the compound can inhibit the activity of DPP-IV, so that the muscle nutrition related protein level in muscles is up-regulated, and the integral improvement of muscle functions is realized. Embodiments herein also result in a composition for upregulating muscle nutrition related protein levels in muscle cells.

Described herein are methods of treating muscular dystrophy comprising administering a recombinant AAV (rAAV) scAAVrh74.MHCK7.hSGCB vector, methods of expressing beta-sarcoglycan gene in a patient, pharmaceutical compositions comprising the rAAV, and methods of generating the rAAV.

Disclosed herein are compositions that are useful for targeting a signaling pathway modulator to a muscle cell. In some instances, such compositions may be useful in treating a muscle disorder, such as muscular dystrophy.

Stabilized amorphous calcium carbonate (ACC) for treatment of several neurological, muscular and infertility diseases and conditions is provided. In particular, the stabilized ACC may be used in the treatment of axonal defects and muscular dystrophy. In addition, provided are improved methods used in assistant reproductive technology. Examples of such methods are in vitro fertilization and improvement of sperm quality. The improved IVF method, for example, comprises addition of the stabilized ACC to the cell culture medium in which the stages of fertilization and embryo development occurs.

The disclosure provides for methods of assessing the risks (e.g., associated with a gene therapy for the treatment of DMD (e.g., delandistrogene moxeparvovec) comprising genotyping the DMD gene and analyzing the HLA type of a subject in need of the gene therapy (e.g., delandistrogene moxeparvovec).

The technology described herein is directed to methods and compositions relating to chimeric polypeptides, e.g., for treatment of Duchenne muscular dystrophy.

Provided are methods of treating or ameliorating the symptoms of limb girdle muscular dystrophy by administration of therapeutically effective doses of adeno-associated (AAV) or recombinant adeno-associated viruses (rAAV) containing a transgene encoding AU- rich element-binding factor 1 (AUF1) effective to treat the limb girdle muscular dystrophy. Also provided are AAV and rAAV vectors encoding AUF1 proteins.

The present invention provides oligonucleotides useful for the treatment of Duchenne muscular dystrophy or Becker muscular dystrophy, compositions containing oligonucleotides, and methods for prevention, treatment, and / or delay. [Solution] An oligonucleotide is provided that comprises a 2'-O-methylRNA monomer and a phosphorothioate skeleton, and also comprises 5-methyluracil and / or 5-methylcytosine and / or a 2,6-diaminopurine base.

This invention relates to adjustable carrying capacity COL6A1 The invention relates to an oligonucleotide expressing a dominant mutant allele, wherein the downregulation is achieved by hybridization of the oligonucleotide with the RNA transcript of the allele at the dominant mutation site, and the oligonucleotide does not inhibit the expression of the wild-type allele, or downregulates the expression of the wild-type allele to a lesser extent than it downregulates the expression of the allele carrying the dominant mutant. The invention also relates to the use of compositions to treat patients with muscular dystrophy, particularly those with malnutrition related to type VI collagen.

The invention relates to a composition and a method for treating muscular dystrophy. In particular, disclosed herein are polynucleic acid molecules, pharmaceutical compositions, and methods for treating muscular dystrophy (DM1).

The application discloses application of papyriquinone in treatment of muscular dystrophy. The inventors find that papyriquinone can be combined with ERRa, activate ERRa and downstream signal pathways, promote muscle cell differentiation, improve muscle function, promote skeletal muscle regeneration, and finally plays a therapeutic role on Duchenne muscular dystrophy.

Compositions are provided that can be used to deliver a tRNA or tRNA derivative to a cell.SOLUTION: The disclosure provides nanoparticle compositions comprising a tRNA and an amino lipid delivery compound. The amino lipid delivery compound can be a dendrimer, dendron, or dendritic lipid, a polymer such as a polyamide or polyester, or a lipid having one or more hydrophobic moieties. In some embodiments, these compositions can be administered to a patient to treat a genetic disease or disorder, such as cystic fibrosis, Duchenne muscular dystrophy, or cancer.SELECTED DRAWING: None

Delivery of alpha-sarcoglycans via adeno-associated virus vectors, and provision of treatment for muscular dystrophy. [Solution] This specification describes a method for treating muscular dystrophy in a subject, the method using a systemic route of administration, approximately 1.0 × 10 12 vg / kg ~ approx. 5.0×10 15 This includes administering the recombinant AAV vector AAVrh74.tMCK.hSCGA at a dose of vg / kg. Further disclosures include methods for doing so in subjects requiring the expression of cells or alpha-sarcoglycan genes, methods for reducing serum CK levels, and methods for increasing alpha-sarcoglycan-positive fibers in the muscle tissue of subjects.

The present invention relates to the prophylactic and / or therapeutic treatment of a disease selected from the group consisting of long-term COVID disease, myalgic encephalomyelitis / chronic fatigue syndrome (ME / CFS) disease, fibromyalgia, dementia, neoplastic disease, autoimmune disease, schizophrenia, epileptic seizure disease, excessive inflammatory disease, chronic inflammatory bowel disease, rheumatic disease, obesity, and the like. Hyperuricemia, diabetes (IDDM / NIDDM), acute viral diseases, amyotrophic lateral sclerosis (ALS), bronchial asthma, atopic allergic disorders, attention deficit disorder (ADD), attention deficit hyperactivity disorder (ADHD), autism spectrum disorders, autoimmune diseases / antibody-related diseases, bipolar affective disorders, celiac disease (CD), chronic obstructive pulmonary disease (COPD), and the like. Chronic inflammatory bowel disease (CED) (i.e. Crohn's disease, ulcerative colitis), cystic fibrosis / cystic fibrosis, depression, endometriosis, polycystic ovarian syndrome, eating disorders, anxiety disorders, addiction disorders, frontotemporal dementia (Pickk's disease), Graves's disease (GD), generalized cardiovascular diseases, Alzheimer's disease, Parkinson's disease, meningitis, multiple sclerosis (MS), and the like. Muscular dystrophy, neurodermatitis, peripheral arterial occlusion disease (PAOD), pneumonia, generalized post-vaccination syndrome and vaccine damage, post-viral infection syndrome, psoriasis, pulmonary arterial hypertension, restless leg syndrome, restrictive pulmonary disease, rheumatoid arthritis (RA), division affective disorder, sepsis, sicca syndrome (SS), scleroderma, and the like. Systemic lupus erythematosus (SLE) and unsatisfactory fertility desires, using an active agent having a nicotinic acetylcholinergic agonist and / or an active agent having a muscarinic acetylcholinergic agonist and / or a muscarinic acetylcholinergic antagonist. This provides a very effective prophylactic and / or therapeutic treatment for these diseases. This prevention or treatment is also readily tolerable, cost effective and easy to apply.

The present disclosure is concerned with piperidinedione compounds, pharmaceutical compositions comprising the compounds, and methods of treating disorders associated with the presence of a premature termination codon such as, for example, cystic fibrosis, Duchenne muscular dystrophy, aniridia, Becker muscular dystrophy, spinal muscular atrophy, Hurler syndrome, hemophilia, epidermolysis bullosa (e.g., dystrophic (DEB) form, junctional (JEB) form). Usher syndrome, and cancer, using the compounds. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present invention.

The present invention relates to methods and compositions for treating muscle diseases and disorders. The invention provides methods for treating muscle myopathies, including muscular dystrophy and cardiomyopathy, by administering stable, long-acting therapeutic agents for vasoactive intestinal peptides. These agents include one or more elastin-like peptides and can be administered at low doses.

An electromyography (EMG) strap and a wearable electronic device are provided. The EMG strap includes a flexible strap body; a flexible printed circuit board (FPCB), housed within the flexible strap body; a surface electromyography (sEMG) sensor assembly, disposed on the FPCB, where the sEMG sensor assembly is exposed from the flexible strap body and is configured to collect an EMG signal and pre-process the EMG signal to obtain a pre-processed analog signal; and a board-to-board (BTB) connector, disposed on the FPCB and electrically connected to the sEMG sensor assembly. The EMG strap can not only help in diagnosing conditions like muscular dystrophy and motor neuron disease but also play a pivotal role in developing advanced prosthetic devices that respond to muscle signals.