7 results about "Muscular Disorders" patented technology

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.

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.

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.

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.

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.