130 results about "Duchenne muscular 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.

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.

The present invention particularly provides anti-Flt-1 antibodies and methods for treating muscular dystrophy, specifically Duchenne muscular dystrophy (DMD). In some embodiments, the methods according to the present invention include administering an effective amount of an anti-Flt-1 antibody or an antigen binding protein thereof to an individual suffering from or susceptible to DMD, such that at least one symptom or feature of DMD is reduced in intensity, severity, or frequency or the onset is delayed.

The present invention provides novel gene therapy compositions for use in treatment of Duchenne Muscular Dystrophy (DMD).

The present invention relates to activin receptor IIA type variants and methods of use thereof. The present invention features a polypeptide comprising an extracellular ActRIIa variant. In some embodiments, the polypeptides of the present invention comprise an extracellular ActRIIa variant fused to an Fc domain monomer or part. The invention also features the treatment of diseases and conditions involving muscle weakness and atrophy using said polypeptides, such as Duchenne muscular dystrophy, facial shoulder brachial muscular dystrophy, inclusion body myositis, amyotrophic lateral sclerosis, sarcopenia; or cancer cachexia; the present invention relates to pharmaceutical compositions and methods for treating a metabolic disease, such as obesity, type 1 diabetes mellitus or type 2 diabetes mellitus.

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.

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 present invention relates to a composition for preventing or treating Duchenne muscular dystrophy (DMD), which contains a miR-499-5p activator. The miR-499-5p activator promotes the expression of miR-499-5p by expressing MMP-1 in myotube cells, and restores fibrous muscle by down-regulating the activation of TGF-β receptors and phosphorylation of smad2 / 3, and therefore can be used for the prevention or treatment of 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.

Provided are methods of treating or ameliorating the symptoms of dystrophinopathies, such as Duchenne muscular dystrophy and Becker muscular dystrophy by administration of therapeutically effective doses of recombinant adeno-associated viruses (rAAV) containing a transgene encoding a microdystrophin comprising a CT domain.

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 relates to recombinant adeno-associated virus (rAAV) delivery of a GALGT2 polynucleotide. The disclosure provides rAAV and methods of using the rAAV for GALGT2 gene therapy of neuromuscular disorders. Exemplary neuromuscular disorders include, but are not limited to, muscular dystrophies such as Duchenne muscular dystrophy, Congenital Muscular Dystrophy 1A and Limb Girdle Muscular Dystrophy 2D.

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.

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.

Disclosed herein are oligonucleotide conjugates and pharmaceutical compositions for inducing multi-exon skipping. In some instances, also disclosed herein are methods of treating a muscular dystrophy, including treating Duchenne muscular dystrophy or Becker muscular dystrophy.

The present invention provides, inter alia, anti-Flt-1 antibodies and methods for the treatment of muscular dystrophy, in particular Duchenne muscular dystrophy (DMD). In some embodiments, a method according to the invention comprises administering to an individual suffering from or susceptible to DMD an effective amount of an anti-Flt-1 antibody or antigen binding protein thereof such that at least one symptom or feature of DMD is reduced in intensity, severity, or frequency or delayed onset.

The present invention relates to a combination of suitable dose of suitable COX-2 inhibitor and Metformin or its suitable pharmaceutically acceptable salts, wherein the dose of Metformin, or its suitable pharmaceutically acceptable salts is selected in the range of 10 mg to 2000 mg. Present invention also provides use of the said combination for the treatment of Duchenne Muscular Dystrophy and Becker Muscular Dystrophy disease.

Neuromuscular diseases, such as Duchenne Muscular Dystrophy (DMD), Bainite Muscular Dystrophy (BMD), Limb-banding Muscular Dystrophy (LGMD), and MecARDL Disease, are treated with compositions comprising substituted pyridazinone compounds.

The present invention relates to activin receptor IIA type variants and methods of use thereof. The present invention features a polypeptide comprising an extracellular ActRIIa variant. In some embodiments, the polypeptides of the present invention comprise an extracellular ActRIIa variant fused to an Fc domain monomer or part. The invention also features the treatment of diseases and conditions involving muscle weakness and atrophy using said polypeptides, such as Duchenne muscular dystrophy, facial shoulder brachial muscular dystrophy, inclusion body myositis, amyotrophic lateral sclerosis, sarcopenia; or cancer cachexia; the present invention relates to pharmaceutical compositions and methods for treating a metabolic disease, such as obesity, type 1 diabetes mellitus or type 2 diabetes mellitus.

The invention provides sup-tRNA for treating Duchenne muscular dystrophy, a recombinant plasmid and a construction method of the sup-tRNA, and relates to the technical field of biological medicines. The sequence of the sup-tRNA for treating the Duchenne muscular dystrophy is as shown in SEQ ID No. 6. According to the application disclosed by the invention, the sup-tRNA is delivered by utilizing MyoAAV of targeted muscle, PTC (Positive Temperature Coefficient) can be recognized in an mdx mouse body, and the expression of dystrophin protein is increased, so that the muscular dystrophin phenotype of the mdx mouse is effectively relieved, and Ribo-seq also proves that the sup-tRNA has relatively high safety. Meanwhile, the strategy also successfully increases the dystrophin protein expression quantity of skeletal muscle precursor cells and myocardial cells from the DMD patient. In addition, the inventor explores the combined use of three small molecule compounds, namely PTC124, CC-90009 and SRI-41315, and the sup-tRNA, so that the read-through efficiency of the sup-tRNA can be improved. In conclusion, the sup-tRNA readthrough therapy is expected to become a new choice for treating DMD and other nonsense mutation diseases.

The present disclosure relates generally to DG9 peptide-conjugated splice modulating therapies for the treatment of Duchenne muscular dystrophy and spinal muscular atrophy.

The invention discloses application of a Duchenne muscular dystrophy drug in attention deficit and hyperactivity disorder and a product, and belongs to the technical field of bioengineering. Through a zebra fish attention deficit and hyperactivity disorder model, the inventor team finds that the Duchenne muscular dystrophy medicine has the effects of improving attention deficit and hyperactivity disorder diseases and has statistical significance. Therefore, when the Duchenne muscular dystrophy medicine is applied to preparation of products for treating attention deficit and hyperactivity disorder, a reliable theoretical basis is provided for a new treatment scheme.

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.

Some aspects of the present disclosure relate to complexes, and other aspects relate to formulations (e.g., aqueous form, lyophilized form) comprising such complexes (e.g., wherein each complex has an exemplary formula shown below) that comprise a phosphodiamide morpholino oligomer (e.g., an anti-TfR1 antibody) covalently linked to an antibody (e.g., an anti-TfR1 antibody). The method can be used for targeting DMD). The use of these formulations for treating a subject having a mutated DMD allele associated with Dische internal muscular dystrophy is also provided. I) # imgabs0 #

Provided herein are methods of treating or delaying the onset of Duchenne muscular dystrophy using modified antisense oligonucleotides.

The present disclosure is concerned with substituted uracil 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.