Search Results - muscular+disorders

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Combinations of ASOs for the Modulation of SMN Expression in SMA Patients

Unmet NeedSpinal muscular atrophy (SMA) is a devastating neuromuscular disease that predominantly affects children and is the most common cause of hereditary infant mortality. The disease is caused by low levels of survival motor neuron (SMN) protein, due to recessive mutations in the SMN1 gene. Importantly, the copy number variation of SMN2, a homologous...

Published: 3/13/2026 | Inventor(s): Charlotte Sumner, Constantin d'Ydewalle, Frank Rigo, C. Bennett

Keywords(s): CNS and Neurological Disorders, Combination, Disease Indication, Drug Delivery Vehicle, Muscle Atrophy, Muscular Disorders, Neurodegenerative Diseases, Peptides/Prodrugs, Single, Targeted Therapy, Therapeutic Matter, Therapeutics, Therapy Type

Category(s): Clinical and Disease Specializations > Neurology > Motor Neuron Diseases, Technology Classifications > Therapeutic Modalities > Therapeutic Delivery Platforms, Technology Classifications > Therapeutic Modalities > Proteins

Human IPSC-Based Drug Testing Platform for Duchenne Muscular Dystrophy

Unmet NeedThe development of new drugs is a capital and time intensive process. For this reason, the development of orphan drugs for those diseases that affect a small subset of the population. One of such diseases, Duchenne’s muscular dystrophy (DMD) affects 1 in 5000 boys worldwide and has no known cure. Current drug screen platforms include animal...

Published: 3/13/2026 | Inventor(s): Gabsang Lee, Kathryn Wagner, Congshan (Sunny) Sun

Keywords(s): Discovery/Research Tools, Disease Indication, In Vitro Research Tool, Muscular Disorders, Small Molecules, Therapeutic Matter, Therapeutic Substance, Therapeutics

Category(s): Clinical and Disease Specializations > Rare Diseases, Technology Classifications > Therapeutic Modalities > Small Molecules, Technology Classifications > Research Tools > Research Stem Cells

Soluble Form of Activin Type IIB Receptor (ACVR2B/Fc)

Invention Novelty: This technology identifies the use of soluble receptor (ACVR2B/Fc) to block the activities of members of the TGF-beta superfamily of signaling molecules.Value Proposition:Myostatin is a member of the TGF-β family; it suppresses muscle growth and its absence stimulates muscle growth substantially. Most of the myostatin inhibitors...

Published: 3/12/2026 | Inventor(s): Se-jin Lee

Keywords(s): Biologics, Disease Indication, Muscular Disorders, Protein, Therapeutic Matter, Therapeutic Substance, Therapeutics

Category(s): Clinical and Disease Specializations, Clinical and Disease Specializations > Musculoskeletal, Technology Classifications > Therapeutic Modalities > Biologics, Technology Classifications > Therapeutic Modalities > Proteins, Technology Classifications > Therapeutic Modalities

Rabbit Serum 5690

C10529: Rabbit Serum 5690: Antibodies Against Rat Lmo7Value Proposition: • Polyclonal antibodies against rat Lmo7Technical Details: Nuclear lamina is a dense fibrillar network inside the nucleus of eukaryotic cells composed of intermediate filaments (lamins) and membrane associated proteins called lamin-associated membrane proteins. The nuclear lamina...

Published: 3/12/2026 | Inventor(s): Katherine Wilson

Keywords(s): Antibodies, Basic Research Biomarker, Discovery/Research Tools, Disease Indication, Genetic Disorders, Muscular Disorders, Peptides, Polyclonal Antibodies, Proteomics/Immunoassays, Proteomics/Immunoassays Antiserum, Recombinant Proteins, Research Reagent

Category(s): Technology Classifications > Research Tools > Antibodies, Technology Classifications > Research Tools > Assays, Technology Classifications > Research Tools, Technology Classifications > Research Tools > Proteins, Ligands & Receptors, Clinical and Disease Specializations > Genetic Diseases, Clinical and Disease Specializations > Rare Diseases, Clinical and Disease Specializations > Musculoskeletal

C05182: Novel Protein Increases Muscle Mass

Technical Details: Muscle mass is normally regulated by the activities of members of the transforming growth factor-B (TGF-B) superfamily, which act to limit muscle growth. Scientists at JHU have now found a protein that is capable of binding certain members of the TGF-B family and inhibiting their activities in vitro. Therefore this protein has important...

Published: 3/12/2026 | Inventor(s): Se-jin Lee

Keywords(s): Biologics, Disease Indication, Muscular Disorders, Protein, Single, Targeted Therapy, Therapeutic Matter, Therapeutic Substance, Therapeutics, Therapy Type