Search Results - rare+and+orphan+disease+treatments

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A GALT-null Rat Model for Studies of Classic Galactosemia
Application Pre-clinical rodent model to study classic galactosemia disease. Key Benefits Identify striking differences in GALT-independent galactose metabolism among tissues and across post-natal time points. Study the relationship between galactose metabolite levels in blood and other tissues. Study the impact of rigorous galactose restriction...
Published: 2/13/2025   |   Inventor(s): Judith Fridovich-Keil
Keywords(s): Animal Model, Rare and Orphan Disease Treatments
Category(s): TechPub Algolia > Research Tools
Novel Therapeutic Target for Fragile X Syndrome and Neurodevelopmental Disorders
Application Salvianolic acid C for treating Fragile X Syndrome (FXS). Key Benefits New therapeutic drug candidate for the treatment of FXS. Preliminary data in the laboratory in organoids treated with the drug showed rescued cell phenotypes known to be associated with the disease in humans. Potential to treat other neurodevelopmental disorders. Market...
Published: 2/13/2025   |   Inventor(s): Peng Jin, Chuan He, Zhongyu Zou
Keywords(s): Neurology and Pain Treatments, Rare and Orphan Disease Treatments
Category(s): TechPub Algolia > Therapeutics
Small Molecules for Treating Cystic Fibrosis
ApplicationSmall molecule compounds to potentiate most common type of CFTR mutation in cystic fibrosis patients.Key BenefitsPotentiates ΔF508-hCFTR mutation, addressing more than 60 percent of CF patients. Market SummaryCystic fibrosis transmembrane conductance regulator (CFTR) is a membrane protein and ion channel that malfunctions in the genetic...
Published: 2/13/2025   |   Inventor(s): Nael McCarty, Guiying Cui, Hanoch Senderowitz, Netaly Khazanov
Keywords(s): Rare and Orphan Disease Treatments, Respiratory Treatments, Small Molecule
Category(s): TechPub Algolia > Therapeutics, TechPub Algolia > Drug Discovery
Antisense Oligonucleotides Therapeutics Targeting Specific Tissues or Cells
Application Antisense oligonucleotide (ASO) therapies for the treatment of rare diseases and cancer. Key Benefits Novel approach to ASO therapeutics that potentially improve tissue/cell targeting capabilities. Increases the efficacy of ASOs while decrease off-target toxicity. Potential to treat a variety of genetic diseases such as cancer, blood...
Published: 2/13/2025   |   Inventor(s): Jiahui Zhang, Khalid Salaita, Hanjoong Jo
Keywords(s): Hematology and Blood Products, Oncology Treatments, Platform, Rare and Orphan Disease Treatments
Category(s): TechPub Algolia > Therapeutics
Predicting Treatment Response in Patients with Spinal Muscular Atrophy (SMA)
Application Prognostic model predicts treatment response to nusinersen (Spinraza®) in patients with spinal muscular atrophy. Key Benefits A prediction model using baseline CHOP-INTEND, IL-8, fractalkine, and MCP-1 accurately predicts CHOP-INTEND outcomes in children treated with nusinersen The model can also be incorporated into future therapeutic...
Published: 2/13/2025   |   Inventor(s): William Hu, Sumit Verma
Keywords(s): Biomarkers, Musculoskeletal Treatments, Pediatrics, Rare and Orphan Disease Treatments
Category(s): TechPub Algolia > Diagnostics
CFTR Modulators for Treating Rare Forms of Cystic Fibrosis
Application Small molecules to treat cystic fibrosis and other pulmonary diseases. Key Benefits Rescue rare variants of CFTR gene. Serve as PDE4 inhibitors with strong anti-inflammatory effects. Market Summary Cystic fibrosis (CF) is a progressive, genetic disorder that arises from mutations in the cystic fibrosis transmembrane conductance...
Published: 3/6/2025   |   Inventor(s): Eric J. Sorscher, Jeong Hong, Haian Fu, Yuhong Du, Huw Davies, Andras Rab, Candela Manfredi, Xun Yang, Zhi Ren
Keywords(s): Rare and Orphan Disease Treatments, Respiratory Treatments, Small Molecule
Category(s): TechPub Algolia > Therapeutics
Assay for Non-Nucleoside Inhibitors of the Measles and Nipah Viruses
Application A cell-based assay to screen for inhibitors of the measles virus (MV). Highly active compounds have been identified and analogs have been synthesized and evaluated. Key Benefits Potent (sub-nM range), non-peptidic, non-nucleosidic compounds. Robust, cell-based, high-throughput screening assay. Technical Summary Vaccination serves...
Published: 2/13/2025   |   Inventor(s): Richard Plemper, James Snyder, Aiming Sun
Keywords(s): Microbiology and Infectious Disease Treatments, Rare and Orphan Disease Treatments, Screening Assays
Category(s): TechPub Algolia > Therapeutics, TechPub Algolia > Drug Discovery, TechPub Algolia > Research Tools