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354 results about "Muscular dystrophy" patented technology
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A group of inherited conditions affecting the muscles, gradually leading to disability.
Oligomers
ActiveUS20100168212A1High degreeOrganic active ingredientsSugar derivativesMuscular dystrophyOligomer
Molecules are provided for inducing or facilitating exon skipping in forming spliced mRNA products from pre-mRNA molecules in cells. The molecules may be provided directly as oligonucleotides or expression products of vectors that are administered to a subject. High rates of skipping can be achieved. High rates of skipping reduce the severity of a disease like Duchene Muscular Dystrophy so that the disease is more like Becker Muscular Dystrophy. This is a severe reduction in symptom severity and mortality.
Owner:ROYAL HOLLOWAY & BEDFORD NEW COLLEGE
Modified and stabilized GDF propeptides and uses thereof
InactiveUS7202210B2Prevent practical therapeuticPrevent prophylactic utilityFungiBacteriaAcute hyperglycaemiaMuscle tissue
Modified and stabilized propeptides of Growth Differentiation Factor proteins, such as GDF-8 and Bone Morphogenetic Protein-11, are disclosed. Also disclosed are methods for making and using the modified propeptides to prevent or treat human or animal disorders in which an increase in muscle tissue would be therapeutically beneficial. Such disorders include muscle or neuromuscular disorders (such as amyotrophic lateral sclerosis, muscular dystrophy, muscle atrophy, congestive obstructive pulmonary disease, muscle wasting syndrome, sarcopenia, or cachexia), metabolic diseases or disorders (such as such as type 2 diabetes, noninsulin-dependent diabetes mellitus, hyperglycemia, or obesity), adipose tissue disorders (such as obesity), and bone degenerative diseases (such as osteoporosis).
Owner:WYETH LLC
Oligomers
Molecules are provided for inducing or facilitating exon skipping in forming spliced mRNA products from pre-mRNA molecules in cells. The molecules may be provided directly as oligonucleotides or expression products of vectors that are administered to a subject. High rates of skipping can be achieved. High rates of skipping reduce the severity of a disease like Duchene Muscular Dystrophy so that the disease is more like Becker Muscular Dystrophy. This is a severe reduction in symptom severity and mortality.
Owner:ROYAL HOLLOWAY & BEDFORD NEW COLLEGE
Lower limb walking external skeleton capable of being worn
InactiveCN1586434AEnhance walking abilityReduce fatigueArtificial legsMuscular dystrophyHuman motion
The wearable lower limb walking external skeleton consists of waist support, four-rod hip mechanism, four-rod knee mechanism, four-rod heel knee mechanism and planta support connected successively. For amputated patient or muscular dystrophy patient, the present invention has auxiliary program written in advance for assisting walking. For tourist and the weak, the present invention is worn and the wearer walks, so that the displacement sensors acquires the motion gesture signals of the wearer, and the gait is recorded in the computer and copied to strengthen the walking ability of the wearer. The present invention is used as the walking aid for the patients and the equipment for walking tourist.
Owner:ZHEJIANG UNIV
Modified and stabilized gdf propeptides and uses thereof
InactiveUS20070149455A1Avoid utilizationImproved pharmacokinetic propertiesFungiBacteriaMuscle tissueAmytrophic lateral sclerosis
Modified and stabilized propeptides of Growth Differentiation Factor proteins, such as GDF-8 and Bone Morphogenetic Protein-11, are disclosed. Also disclosed are methods for making and using the modified propeptides to prevent or treat human or animal disorders in which an increase in muscle tissue would be therapeutically beneficial. Such disorders include muscle or neuromuscular disorders (such as amyotrophic lateral sclerosis, muscular dystrophy, muscle atrophy, congestive obstructive pulmonary disease, muscle wasting syndrome, sarcopenia, or cachexia), metabolic diseases or disorders (such as such as type 2 diabetes, noninsulin-dependent diabetes mellitus, hyperglycemia, or obesity), adipose tissue disorders (such as obesity), and bone degenerative diseases (such as osteoporosis).
Owner:WYETH LLC
Gel-based delivery of recombinant adeno-associated virus vectors
InactiveUS20060078542A1High transduction efficiencyIncrease exposure timeBiocidePowder deliveryMuscle tissueHeart disease
Disclosed are water-soluble gel-based compositions for the delivery of recombinant adeno-associated virus (rAAV) vectors that express nucleic acid segments encoding therapeutic constructs including peptides, polypeptides, ribozymes, and catalytic RNA molecules, to selected cells and tissues of vertebrate animals. Also disclosed are gel-based rAAV compositions are useful in the treatment of mammalian, and in particular, human diseases, including for example, cardiac disease or dysfunction, and musculoskeletal disorders and congenital myopathies, including, for example, muscular dystrophy, acid maltase deficiency (Pompe's disease), and the like. In illustrative embodiments, the invention provides rAAV vectors comprised within a biocompatible gel composition for enhanced viral delivery / transfection to mammalian tissues, and in particular to vertebrate muscle tissues such as a human heart or diaphragm tissue.
Owner:UNIV OF FLORIDA RES FOUNDATION INC
Automated therapy table for treating lower extremities and method therefor
An automated therapy table is disclosed. The automated therapy table may have various support portions capable of independent automatic actuation of a person's lower extremities through passive exercise. The automated therapy table allows a patient to perform leg elevation, approximation / decompression of the leg, internal / external rotation of the leg, ankle plantar flexion / dorsiflexion, and foot inversion / eversion movements. During each movement, the patient may be instructed to think in the direction of the movement. It has been found that doing so helps increase the healing effects. The disclosed table and method may be beneficial for patients after certain operations as well as for those suffering from various forms of debilitating illnesses, such as Multiple Sclerosis, Charcot-Marie-Tooth, and Muscular Dystrophy.
Owner:SCHAEFFER DWIGHT L
Exon skipping compositions for treating muscular dystrophy
InactiveUS20140315977A1Enhance the activity, cellular distribution, or cellular uptake of the antisense oligonucleotideOrganic active ingredientsSplicing alterationDuchenne muscular dystrophyMuscular dystrophy
Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 53 skipping are described.
Owner:SAREPTA THERAPEUTICS INC
Exon skipping compositions for treating muscular dystrophy
InactiveUS20160040162A1Enhance the activity, cellular distribution, or cellular uptake of the antisense oligonucleotideOrganic active ingredientsSplicing alterationDuchenne muscular dystrophyMuscular dystrophy
Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 53 skipping are described.
Owner:SAREPTA THERAPEUTICS INC
Novel agents for preventing and treating disorders involving modulation of the RyR receptors
InactiveUS20070049572A1Improve bindingLow affinityBiocideOrganic active ingredientsSIDS - Sudden infant death syndromeDisease cause
The present invention provides novel compounds of Formula I and salts, hydrates, solvates, complexes, and prodrugs thereof. The present invention further provides methods for synthesizing compounds of Formula I. The invention additionally provides pharmaceutical compositions comprising the compounds of Formula I and methods of using the pharmaceutical compositions of Formula I to treat and prevent disorders and diseases associated with the RyR receptors that regulate calcium channel functioning in cells. Such disorders and diseases include, by way of example only, cardiac disorders and diseases, skeletal muscular disorders and diseases, cognitive disorders and diseases, malignant hyperthermia, diabetes, and sudden infant death syndrome. Cardiac disorder and diseases include, but are not limited to, irregular heartbeat disorders and diseases; exercise-induced irregular heartbeat disorders and diseases; sudden cardiac death; exercise-induced sudden cardiac death; congestive heart failure; chronic obstructive pulmonary disease; and high blood pressure. Irregular heartbeat disorders and diseases include and exercise-induced irregular heartbeat disorders and diseases include, but are not limited to, atrial and ventricular arrhythmia; atrial and ventricular fibrillation; atrial and ventricular tachyarrhythmia; atrial and ventricular tachycardia; catecholaminergic polymorphic ventricular tachycardia (CPVT); and exercise-induced variants thereof. Skeletal muscular disorder and diseases include, but are not limited to, skeletal muscle fatigue, exercise-induced skeletal muscle fatigue, muscular dystrophy, bladder disorders, and incontinence. Cognitive disorders and diseases include, but are not limited to, Alzheimer's Disease, forms of memory loss, and age-dependent memory loss.
Owner:THE TRUSTEES OF COLUMBIA UNIV IN THE CITY OF NEW YORK
Exon skipping compositions for treating muscular dystrophy
InactiveUS20140329881A1Enhance the activity, cellular distribution, or cellular uptake of the antisense oligonucleotideOrganic active ingredientsSplicing alterationDuchenne muscular dystrophyMuscular dystrophy
Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 44 skipping are described.
Owner:SAREPTA THERAPEUTICS INC
Exon skipping compositions for treating muscular dystrophy
InactiveUS20150361428A1Enhance the activity, cellular distribution, or cellular uptake of the antisense oligonucleotideSplicing alterationSugar derivativesDuchenne muscular dystrophyMuscular dystrophy
Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 53 skipping are described.
Owner:SAREPTA THERAPEUTICS INC
Novel agents for preventing and treating disorders involving modulation of the RyR receptors
ActiveUS20060194767A1BiocideOrganic active ingredientsSIDS - Sudden infant death syndromeCognitive disorder
The present invention provides novel compounds of Formula I and salts, hydrates, solvates, complexes, and prodrugs thereof. The present invention further provides methods for synthesizing compounds of Formula I. The invention additionally provides pharmaceutical compositions comprising the compounds of Formula I and methods of using the pharmaceutical compositions of Formula I to treat and prevent disorders and diseases associated with the RyR receptors that regulate calcium channel functioning in cells. Such disorders and diseases include, by way of example only, cardiac disorders and diseases, skeletal muscular disorders and diseases, cognitive disorders and diseases, malignant hyperthermia, diabetes, and sudden infant death syndrome. Cardiac disorder and diseases include, but are not limited to, irregular heartbeat disorders and diseases; exercise-induced irregular heartbeat disorders and diseases; sudden cardiac death; exercise-induced sudden cardiac death; congestive heart failure; chronic obstructive pulmonary disease; and high blood pressure. Irregular heartbeat disorders and diseases include and exercise-induced irregular heartbeat disorders and diseases include, but are not limited to, atrial and ventricular arrhythmia; atrial and ventricular fibrillation; atrial and ventricular tachyarrhythmia; atrial and ventricular tachycardia; catecholaminergic polymorphic ventricular tachycardia (CPVT); and exercise-induced variants thereof. Skeletal muscular disorder and diseases include, but are not limited to, skeletal muscle fatigue, exercise-induced skeletal muscle fatigue, muscular dystrophy, bladder disorders, and incontinence. Cognitive disorders and diseases include, but are not limited to, Alzheimer's Disease, forms of memory loss, and age-dependent memory loss.
Owner:THE TRUSTEES OF COLUMBIA UNIV IN THE CITY OF NEW YORK
Fibronectin based scaffold domain proteins that bind to myostatin
ActiveUS8853154B2Inhibitory activityIncrease volumeBacteriaPeptide/protein ingredientsMyostatinFibrosis
The present invention relates to fibronectin-based scaffold domain proteins that bind to myostatin. The invention also relates to the use of these proteins in therapeutic applications to treat muscular dystrophy, cachexia, sarcopenia, osteoarthritis, osteoporosis, diabetes, obesity, COPD, chronic kidney disease, heart failure, myocardial infarction, and fibrosis. The invention further relates to cells comprising such proteins, polynucleotides encoding such proteins or fragments thereof, and to vectors comprising the polynucleotides encoding the proteins.
Owner:BRISTOL MYERS SQUIBB CO
Compositions and Methods for Treating and Preventing Inflammatory and/or Degenerative Processes in Humans and Other Animals
Disclosed are compositions useful for treating Alzheimer's disease, atherosclerosis, arteriosclerosis, osteoarthritis and other degenerative joint diseases, Huntington's chorea, Parkinson's disease, optic atrophy, retinitis pigmentosa, macular degeneration, muscular dystrophy, aging-associated degenerative processes, asthma, dermatitis, laminitis, pemphigoid, pemphigus, reactive airway disease (e.g., COPD, IAD), inflammatory bowel disease (e.g., Crohn's disease, ulcerative colitis), multiple sclerosis, rheumatoid arthritis, periodontal disease, systemic lupus erythematosus, sarcoidosis, psoriasis, type I diabetes, ischemia-reperfusion injury, chronic inflammatory diseases, geriatric wasting, cancer cachexia, cachexia associated with chronic inflammation, sick feeling syndrome, and other inflammatory and / or degenerative diseases, disorders, conditions, and processes in humans and other animals. In one embodiment, the compositions include at least 4 of the following: a MMP1 inhibitor, a MMP2 inhibitor, a MMP3 inhibitor, a MMP7 inhibitor, a MMP9 inhibitor, an ADAMTS-4 inhibitor, a MMP13 inhibitor, and a MMP14 inhibitor. In another embodiment, the compositions include a curcuminoid, a polymethoxylated flavone, a catechin, and a boswellic acid.
Owner:BAKER DONALD J
Polymorphs of an androgen receptor modulator
InactiveUS20070129548A1Maintain good propertiesEasy to processOrganic active ingredientsSkeletal disorderDiseaseAging skins
Compounds of structural formula I are modulators of the androgen receptor (AR) in a tissue selective manner. These compounds are useful in the enhancement of weakened muscle tone and the treatment of conditions caused by androgen deficiency or which can be ameliorated by androgen administration, including osteoporosis, osteopenia, glucocorticoid-induced osteoporosis, periodontal disease, bone fracture, bone damage following bone reconstructive surgery, sarcopenia, frailty, aging skin, male hypogonadism, postmenopausal symptoms in women, atherosclerosis, hypercholesterolemia, hyperlipidemia, obesity, aplastic anemia and other hematopoietic disorders, inflammatory arthritis and joint repair, HIV-wasting, prostate cancer, benign prostatic hyperplasia (BPH), abdominal adiposity, metabolic syndrome, type II diabetes, cancer cachexia, Alzheimer's disease, muscular dystrophies, cognitive decline, sexual dysfunction, sleep apnea, depression, premature ovarian failure, and autoimmune disease, alone or in combination with other active agents.
Owner:MERCK SHARP & DOHME CORP
17-heterocyclic-4-azasteroid derivatives as androgen receptor modulators
Compounds of structural formula I are modulators of the androgen receptor (AR) in a tissue selective manner. They are useful as agonists of the androgen receptor in bone and / or muscle tissue while antagonizing the AR in the prostate of a male patient or in the uterus of a female patient. These compounds are therefore useful in the enhancement of weakened muscle tone and the treatment of conditions caused by androgen deficiency or which can be ameliorated by androgen administration, including osteoporosis, osteopenia, glucocorticoid-induced osteoporosis, periodontal disease, bone fracture, bone damage following bone reconstructive surgery, sarcopenia, frailty, aging skin, male hypogonadism, postmenopausal symptoms in women, atherosclerosis, hypercholesterolemia, hyperlipidemia, obesity, aplastic anemia and other hematopoietic disorders, inflammatory arthritis and joint repair, HIV-wasting, prostate cancer, cancer cachexia, Alzheimer's disease, muscular dystrophies, cognitive impairment, decreased libido, premature ovarian failure, and autoimmune disease, alone or in combination with other active agents.
Owner:MERCK SHARP & DOHME CORP
Automated therapy table for treating lower extremities and method therefor
An automated therapy table is disclosed. The automated therapy table may have various support portions capable of independent automatic actuation of a person's lower extremities through passive exercise. The automated therapy table allows a patient to perform leg elevation, approximation / decompression of the leg, internal / external rotation of the leg, ankle plantar flexion / dorsiflexion, and foot inversion / eversion movements. During each movement, the patient may be instructed to think in the direction of the movement. It has been found that doing so helps increase the healing effects. The disclosed table and method may be beneficial for patients after certain operations as well as for those suffering from various forms of debilitating illnesses, such as Multiple Sclerosis, Charcot-Marie-Tooth, and Muscular Dystrophy.
Owner:SCHAEFFER DWIGHT L
Ultrasonic Characterization of Internal Body Conditions Using Information Theoretic Signal Receivers
InactiveUS20070232909A1Enhance the imageAdvance in genomics and proteomicsUltrasonic/sonic/infrasonic diagnosticsDispersion deliveryMuscular dystrophyMedical imaging
Disclosed herein is a technique for performing medical imaging on a region of interest (ROI) wherein information theoretic signal receivers are used to enhance the detection and monitoring of pathologies such as angiogenesis and muscular dystrophy. Examples of information theoretic signal receivers that can be used in the practice of the invention include Shannon entropy signal receivers, continuous limit Shannon entropy signal receivers, Renyi entropy signal receivers, specific heat analog signal receivers, and thermodynamic energy analog signal receivers. The contrast enhancement provided by contrast agents, either targeted contrast agents or bubble-based contrast agents, is enhanced through the use of such information theoretic signal receivers. Further still, the contrast agents can be heated to further enhance visualization with information theoretic signal receivers.
Owner:WASHINGTON UNIV IN SAINT LOUIS
Fluorinated 4-azasteroid derivatives as androgen receptor modulators
Compounds of structural formula I are modulators of the androgen receptor (AR) in a tissue selective manner. They are useful as agonists of the androgen receptor in bone and / or muscle tissue while antagonizing the AR in the prostate of a male patient or in the uterus of a female patient. These compounds are therefore useful in the treatment of conditions caused by androgen deficiency or which can be ameliorated by androgen administration, including osteoporosis, osteopenia, glucocorticoid-induced osteoporosis, periodontal disease, bone fracture, bone damage following bone reconstructive surgery, sarcopenia, frailty, aging skin, male hypogonadism, postmenopausal symptoms in women, atherosclerosis, hypercholesterolemia, hyperlipidemia, obesity, aplastic anemia and other hematopoietic disorders, inflammatory arthritis and joint repair, HIV-wasting, prostate cancer, cancer cachexia, muscular dystrophies, premature ovarian failure, and autoimmune disease, alone or in combination with other active agents.
Owner:MERCK SHARP & DOHME CORP
N-(2-benzyl)-2-phenylbutanamides as androgen receptor modulators
InactiveUS20050277681A1Minimal effectReduce decreaseBiocideNervous disorderGlucocorticoidAutoimmune disease
Compounds of structural formula I are modulators of the androgen receptor (AR) in a tissue selective manner. These compounds are useful in the enhancement of weakened muscle tone and the treatment of conditions caused by androgen deficiency or which can be ameliorated by androgen administration, including osteoporosis, osteopenia, glucocorticoid-induced osteoporosis, periodontal disease, bone fracture, bone damage following bone reconstructive surgery, sarcopenia, frailty, aging skin, male hypogonadism, postmenopausal symptoms in women, atherosclerosis, hypercholesterolemia, hyperlipidemia, obesity, aplastic anemia and other hematopoietic disorders, inflammatory arthritis and joint repair, HIV-wasting, prostate cancer, benign prostatic hyperplasia (BPH), abdominal adiposity, metabolic syndrome, type II diabetes, cancer cachexia, Alzheimer's disease, muscular dystrophies, cognitive decline, sexual dysfunction, sleep apnea, depression, premature ovarian failure, and autoimmune disease, alone or in combination with other active agents.
Owner:MERCK SHARP & DOHME CORP
Medicine for treating muscular dystrophy and myasthenia gravis, and its prepn. method
ActiveCN1785223ASignificant effectMuscular disorderNeuromuscular disorderPrimary motor neuronPharmacology
A Chinese medicine for treating the myophagism and myasthenia gravis caused by motor neuron diseases, prograssive myodystrophy and congenital myopathy is prepared from ginseng and epimedium. Its preparing process is also disclosed.
Owner:SHIJIAZHUANG YILING PHARMA
N-(2-benzyl)-2-phenylbutanamides as androgen receptor modulators
Owner:MERCK SHARP & DOHME CORP
Cardiosphere-derived cells and exosomes secreted by such cells in the treatment of muscular dystrophy
ActiveUS20170290860A1Reduce fibrosisImprove mitochondrial functionOrganic active ingredientsSpecial deliveryTissue repairCell free
Described herein are compositions and techniques related to generation and therapeutic application of cardiosphere-derived cells (CDCs) and CDC-derived exosomes. These cells and their secreted vesicles contain a unique milieu of biological factors, including cytokines, growth factors, transcription factors, nucleic acids including non-coding nucleic acids such as microRNAs, that serve to initiate and promote many therapeutic effects. Exosomes and their “cargo” contents, such as microRNAs can favorably modulate apoptosis, inflammation and fibrosis in the injured heart. Thus, CDC-derived exosomes represent a novel “cell-free” therapeutic candidate for tissue repair.
Owner:CEDARS SINAI MEDICAL CENT
ActRIIB proteins and variants and uses therefore relating to utrophin induction for muscular dystrophy therapy
Owner:ACCELERON PHARMA INC
Antisense Polynucleotides to Induce Exon Skipping and Methods of Treating Dystrophies
ActiveUS20150225718A1Inhibit progressImprove muscle functionSplicing alterationSugar derivativesDiseasePolynucleotide
Antisense polynucleotides and their use in pharmaceutical compositions to induce exon skipping in targeted exons of the gamma sarcoglycan gene are provided, along with methods of preventing or treating dystrophic diseases such as Limb-Girdle Muscular Dystrophy. One aspect the disclosure provides an isolated antisense polynucleotide wherein the polynucleotide specifically hybridizes to an exon target region of a gamma sarcoglycan RNA, wherein the exon is selected from the group consisting of exon 4 (SEQ ID NO:1), exon 5 (SEQ ID NO: 2), exon 6 (SEQ ID NO: 3), exon 7 (SEQ ID NO: 4) and a combination thereof. In some embodiments, the antisense polynucleotide cannot form an RNase H substrate, and in further embodiments the antisense polynucleotide comprises a modified polynucleotide backbone.
Owner:UNIVERSITY OF CHICAGO
Novel cyclosporin derivatives and uses thereof
The present invention relates to a compound of the Formula (I)):or pharmaceutically acceptable salt thereof, wherein the symbols are as defined in the specification; a pharmaceutical composition comprising the same, a method for treating or preventing viral infections, inflammation, dry eye, central nervous disorders, cardiovascular diseases, cancer, obesity, diabetes, muscular dystrophy, and hair loss.
Owner:S&T GLOBAL
Lipid raft, caveolin protein, and caveolar function modulation compounds and associated synthetic and therapeutic methods
The present invention is directed to the modulation of lipid rafts, caveolin proteins, or caveolar functions and processes by platinum(IV) compounds. Caveolae and / or lipid rafts are associated with cell transcription regulation, membrane and cellular transport, cell membrane receptor function, cellular trafficking, antigen presentation, cell differentiation and activation, cytokine modulation, membrane structure and function, and protein modulation. Caveolae, caveolin proteins and lipid rafts are known therapeutic targets for numerous biological functions. Diseases and disorders currently known to be therapeutically targeted through caveolae and / or lipid rafts include diabetes, cancer, cardiovascular diseases, atherosclerosis, pulmonary fibrosis, multiple sclerosis, viral and prion diseases, neuronal disorders, degenerative muscular dystrophies, and autoimmune disorders.
Owner:KAY HEIDI
Devices and processes for distribution of genetic material to mammalian limb
InactiveUS20070244067A1Improve breathabilityAltering endogenous propertyFermentationGene therapyMuscular dystrophyMedicine
A process is described for the delivery of a therapeutic polynucleotide to limb muscle tissue suffering from or potentially suffering from Muscular Dystrophy. The polynucleotide is inserted into a mammalian limb vessel such as an artery. Delivery efficiency and distribution is enhanced by combining injection of a solution containing the polynucleotide with the use of an externally applied cuff.
Owner:ARROWHEAD MADISON
Methods and materials for producing transgenic artiodactyls
ActiveUS20120220037A1Animal cellsCell receptors/surface-antigens/surface-determinantsBiological materialsHuman disease
Swine animal models comprising a genomic disruption of an endogenous gene chosen from the group consisting of a Low-Density Lipoprotein Receptor gene LDLR, Duchene's Muscular Dystrophy (DMD) gene, and hairless gene (HR). Methods of preparing transfected cells useful for making a transgenic animal comprising exposing a first group of cells to a transfection agent and reseeding the group with additional cells that have not been exposed to the agent. The transgenic animals are useful for medical and scientific animal models of human diseases and conditions, as well as sources for cells, tissues, and biomaterials.
Owner:RECOMBINETICS INC
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