17 results about "Muscular Disorders" patented technology

The present invention relates to an albumin nanocomposite comprising a phytochemical, and a composition for improving muscle diseases comprising the same, and more specifically, to an albumin nanocomposite comprising a phytochemical, the present invention relates to an albumin nanocomposite containing phytochemicals, and more specifically, to an albumin nanocomposite containing phytochemicals, which exhibits the effects of inhibiting muscle reduction caused by oxidative stress or inflammatory response and promoting the differentiation of myoblasts into muscle cells. The albumin nanocomposite containing the phytochemicals can target and induce immune cells of active oxygen and inflammatory reaction and effectively deliver the phytochemicals, so that muscle cell atrophy is inhibited and differentiation is promoted by adjusting a muscle reduction signal mechanism induced by the active oxygen and the inflammatory reaction, and further muscle reduction can be improved, so that the curative effect of the albumin nanocomposite is improved. The muscular dystrophy is prevented and treated. Therefore, the composition containing the albumin nanocomposite containing phytochemicals of the present invention can be provided as a composition for improving muscle diseases.

Provided herein are compositions, uses thereof and methods for treating muscular dystrophy and / or chronic inflammatory myopathy. The composition comprises at least one compound inhibiting CCR2 signaling and at least one compound inhibiting CSF-1R signaling.

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.

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.

Disclosed herein are substituted pyridazinone compounds, conjugates, and pharmaceutical compositions for treating neuromuscular diseases such as Duchenne Muscular Dystrophy (DMD). The disclosed compounds are particularly useful for treating DMD and modulating the inflammatory inhibitors IL-1, IL-6, or TNF-alpha.

The present invention relates to a gene therapy method capable of continuously and efficiently correcting genes, and a gene therapy product using same. It is expected that the gene therapy product of the present invention can present clues for gene therapy for muscular diseases including Duchenne muscular dystrophy.

This disclosure relates to the use of Purine Nucleoside Phosphorylase (PNP) inhibitors such as ulodesine and its salts, in the treatment and / or prevention of diseases associated with nicotinamide adenine dinucleotide (NAD+) depletion, including diseases of mitochondrial dysfunction (including neurodegeneration and peripheral neuropathies), the preservation of cognitive function and in muscular disorders such as sarcopenia; and in metabolic syndrome and associated conditions. In particular the disclosure provides the use of PNP inhibitors such as ulodesine in the treatment of neurodegenerative conditions such as Parkinson's disease and amyotrophic lateral sclerosis.

The present invention relates to methods and compositions for generating and using myoblast chimeric cells (MCCs) for treating a muscle disease, such as muscular dystrophy, where the MCCs are composed of a myoblast derived from a patient with muscle disease (MD) and a myoblast from a donor without the MD (e.g., a healthy donor). In certain embodiments, cell fusion methods are performed using 2-4, or 5, times passaged myoblasts from the MD and donor subject, and / or polyethylene glycol 0.5-1.5 g / ml. In other embodiments, the MCCs created by fusion are passaged 1-5 times before use, and are passaged at 60-80% confluency. In further embodiments, the myoblasts and / or MCCs are tested at any stage during the process for less than 5-10% CD34 and / or CD45 expression, and / or greater than 50-70% CD56 and / or CD90 expression.

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 substituted pyridazinone compounds, conjugates, and pharmaceutical compositions for treating neuromuscular diseases such as Duchenne Muscular Dystrophy (DMD). The disclosed compounds are particularly useful for treating DMD and modulating the inflammatory inhibitors IL-1, IL-6, or TNF-alpha.

Disclosed is a method of making and using a therapeutically potent cell for treating degenerative muscle disease. More specifically, disclosed is a method of making and using therapeutic cells, the method including identity and potency release assays for selecting an confirming therapeutic cells useful in ameliorating cardiac muscle and / or skeletal muscle degeneration associated with muscular dystrophy.

This invention relates to a method and materials for treating limb-girdle muscular dystrophy R9 (LGMDR9) and congenital muscular dystrophy 1C (MDC1C) using a bicistronic recombinant adeno-associated virus encoding fukutin-related protein (FKRP) and follistatin (FST). This therapy is unique in that it can reconstruct lost muscle strength while simultaneously preventing the subsequent development of muscle disease.

Disclosed is a method of making and using a therapeutically potent cell for treating degenerative muscle disease. More specifically, disclosed is a method of making and using therapeutic cells, the method including identity and potency release assays for selecting an confirming therapeutic cells useful in ameliorating cardiac muscle and / or skeletal muscle degeneration associated with muscular dystrophy.

Human skeletal muscle stem cells were generated from facioscapulohumeral muscular dystrophy (FSHD) and healthy control iPSC using a transgene-free skeletal muscle differentiation protocol and production of stable iMyoblasts. Analyses revealed that FSHD and healthy control iMyoblasts are embryonic-like myogenic cells that undergo myotube differentiation ex vivo by growth factor depletion and are efficiently transplantable into the tibialis anterior (TA) muscles of NSG mice, where human muscle under-goes embryonic-to-adult myosin isoform switching. The DUX4 FSHD disease gene maintains its hypomethylated disease state inFSHD iPSC and iMyoblast, and its expression is upregulated during myotube differentiation and in muscle xenografts. Consequently, these iMyoblasts accurately exhibit the molecular pathology of human muscular dystrophies and are useful for the development of drug, gene editing and stem cell therapeutics.

The present disclosure relates to pharmaceutically acceptable salts and crystalline forms of Compound 1, pharmaceutical compositions comprising the salts and crystalline forms of Compound 1, and their use in treating muscular diseases, such as Duchene Muscular Dystrophy (DMD), that are regulated by AAK1.

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.

Treatments of neuromuscular diseases, such as Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy (BMD), Limb-Girdle Muscular Dystrophy (LGMD), and McArdle's disease, with compositions comprising substituted pyridazinone compounds are described herein.