FMRP

Opna Bio Presents Promising Preclinical Data in Multiple Myeloma with OPN-6602 and in Malignant Mesothelioma with OPN-9840 Showing Significant Tumor Growth Inhibition at the American Association of Cancer Research Annual Meeting

Retrieved on: 
Wednesday, April 10, 2024
Research, Venture Capital, Clinical Trials, Professional Services, Biotechnology, Health, Pharmaceutical, Science, Oncology, CREB-binding protein, NF2, Dexamethasone, FDA, MYC, TGI, Cancer, Bone marrow, Abdomen, Multiple myeloma, Mouse, MYB, Mesothelioma, EP300, Streets of Rage, Doctor of Philosophy, IND, Glomus tumor, TEAD, FMRP, Therapy, BRAF, CBP, Thomas Jefferson University, Patient, Neurofibromatosis, Hippopotamus, Neoplasm, Standard of care, AACR, E1A, IRF4

Data were shared at the American Association of Cancer Research (AACR) Annual Meeting, taking place April 5-10, 2024 in San Diego.

Key Points: 
  • Data were shared at the American Association of Cancer Research (AACR) Annual Meeting, taking place April 5-10, 2024 in San Diego.
  • Multiple myeloma is an aggressive blood cancer derived from malignant plasma cells in the bone marrow.
  • "We are excited to begin our Phase 1 study of OPN-6602 in patients with multiple myeloma this summer.
  • Malignant mesothelioma is a rare and aggressive cancer that primarily affects the lining of the lungs or abdomen.

Orphan designation: Glycyl-L-2-methylprolyl-L-glutamic acid Treatment of fragile X syndrome, 28/07/2015 Positive

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Fragile X syndrome, Civil service commission, Diagnosis, X chromosome, Brain, FMRP, Woman, Learning disability, Disease, Nervous system, Patient, Committee, Knowledge, History, Regulation, Traffic barrier, Anxiety, EMA, IRIS, Mouth, European, COMP, European Commission, Consultant, Safety, Marketing, DSM-IV codes, Turner syndrome, Medicine, Pharmaceutical industry

Orphan designation: Glycyl-L-2-methylprolyl-L-glutamic acid Treatment of fragile X syndrome, 28/07/2015 Positive

Key Points: 


Orphan designation: Glycyl-L-2-methylprolyl-L-glutamic acid Treatment of fragile X syndrome, 28/07/2015 Positive

Zynerba Pharmaceuticals Announces Publication in the Journal of Neurodevelopmental Disorders Describing the Role of the Endocannabinoid System and Cannabidiol Therapy in Fragile X Syndrome

Retrieved on: 
Wednesday, January 11, 2023
CONNECT, Neurodevelopmental disorder, Cannabidiol, Methylation, FMR1, Patient, Fragile X syndrome, Journal, FMRP, ECS, Irritability, Endocannabinoid system, Binary relation, Journal of Neurodevelopmental Disorders, FXS, Medical device, Pharmaceutical industry, Jean-François Zygel

DEVON, Pa., Jan. 11, 2023 (GLOBE NEWSWIRE) -- Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for orphan neuropsychiatric disorders, today announces the publication of a paper entitled, “Role of the Endocannabinoid System in Fragile X Syndrome: Potential Mechanisms for Benefit From Cannabidiol Treatment,” in the Journal of Neurodevelopmental Disorders. The review of evidence suggests a central role for the endocannabinoid system (ECS) in neuronal development and cognitive function and in the pathogenesis of Fragile X syndrome (FXS), and the potential role of cannabidiol as a treatment for FXS. The article can be accessed online at the Journal of Neurodevelopmental Disorders at https://rdcu.be/c25fu.

Key Points: 
  • The review of evidence suggests a central role for the endocannabinoid system (ECS) in neuronal development and cognitive function and in the pathogenesis of Fragile X syndrome (FXS), and the potential role of cannabidiol as a treatment for FXS.
  • The article can be accessed online at the Journal of Neurodevelopmental Disorders at https://rdcu.be/c25fu .
  • “This publication describes the potential of Zygel as a treatment for Fragile X syndrome based on the central role the endocannabinoid system plays in neuronal development and function,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba.
  • Cannabidiol may help restore the function of the ECS in FXS according to a review of data to date.

Lysogene Publishes Positive Preliminary Preclinical Results in the Treatment of Fragile X Syndrome

Retrieved on: 
Thursday, April 21, 2022
Biotechnology, Other Health, Health, Genetics, Clinical Trials, Lists of diseases, Clinical trial, FMR1, ICM, Pathology, Financial Services and Markets Authority (Belgium), CNS, Central nervous system, Brain, Molecular medicine, INSERM, Turner syndrome, Patient, EMBO Molecular Medicine, FXS, MPS, AAV, CSO, Company, Magnetic resonance, FMRP, Forward-looking statement, GM1, Disease, Mutation, Fragile X syndrome, Autism, RNA transfection, CNRS, Adaptive clinical trial, Fragile, Medical device

FXS is caused by a mutation of the FMR1 gene that provides instructions for production of the Fragile X Mental Retardation Protein (FMRP).

Key Points: 
  • FXS is caused by a mutation of the FMR1 gene that provides instructions for production of the Fragile X Mental Retardation Protein (FMRP).
  • Ralph Laufer, CSO of Lysogene, commented: These preclinical results confirm the validity of our innovative approach targeting DGKk for the treatment of FXS, a CNS pathology with high unmet medical need.
  • Lysogene is a gene therapy Company focused on the treatment of orphan diseases of the central nervous system (CNS).
  • Lysogene is also developing an innovative AAV gene therapy approach for the treatment of Fragile X syndrome, a genetic disease related to autism.

Fragile X Syndrome Market Spotlight Report 2021: 10-year Disease Incidence and Prevalence Forecasts, Probability of Success, Licensing and Asset Acquisition Deals, Drug-specific Revenue Forecasts - ResearchAndMarkets.com

Retrieved on: 
Thursday, January 27, 2022
Science, Other Science, Research, Managed Care, General Health, Health, Mental Health, Other Health, FXS, FXTAS, Fragile X syndrome, Turner syndrome, Government budget balance, Novartis, FMRP, Prevalence, FMR1, Therapy, Genetic disorder, Clinical trial, Potassium, Probability, Learning, Mutation, Residential care, Pharmaceutical industry

The "Market Spotlight: Fragile X Syndrome" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Market Spotlight: Fragile X Syndrome" report has been added to ResearchAndMarkets.com's offering.
  • Fragile X syndrome (FXS) is a genetic disorder leading to various developmental problems ranging from learning disabilities to cognitive impairment.
  • FXS is caused by mutation of the fragile X mental retardation 1 (FMR1) gene, which usually makes a protein known as fragile X mental retardation protein (FMRP).
  • Individuals who have other fragile X-associated disorders (FXTAS) have mutations in their FMR1 gene, but they generally make some FMRP.

Zynerba Pharmaceuticals Initiates RECONNECT, a Pivotal Phase 3 Trial of Zygel™ in Fragile X Syndrome

Retrieved on: 
Monday, September 13, 2021
CONNECT, U.S. Securities and Exchange Commission, Security (finance), Synapse, Food, Attention deficit hyperactivity disorder predominantly inattentive, Patient, Mutation, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Form 10-K, Private Securities Litigation Reform Act, Family, FXS, Binary relation, Protein, Marketing, Deviance (sociology), Anxiety, Company, DiGeorge syndrome, Nasdaq, Intellectual, Autism, Clinical Global Impression, Methylation, Annual report, CGG, Safety, DNA, Therapy, Behavior, Ageing, Intellectual disability, Growth, FMRP, COVID-19, Twitter, GLOBE, Fragile X syndrome, Population, ABC, ASD, Infection, Cannabidiol, Pharmaceutical industry, Fine chemical, Residential care

DEVON, Pa., Sept. 13, 2021 (GLOBE NEWSWIRE) -- Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today announces the initiation of a pivotal, multinational randomized, double-blind, placebo-controlled, multiple-center, efficacy and safety (RECONNECT) Phase 3 trial. The RECONNECT trial is designed to evaluate the efficacy and safety of Zygel (cannabidiol formulated in a transdermal gel) in children and adolescents with Fragile X syndrome (FXS). The trial is planned to confirm the positive results observed in a population of responders in the Company’s CONNECT-FX trial, a randomized, double-blind, placebo-controlled trial which assessed the efficacy and safety of Zygel as a treatment for the behavioral symptoms of FXS. FXS is a genetic condition that causes intellectual disability, behavioral and learning challenges and is the most common known single-gene cause of autism spectrum disorder.

Key Points: 
  • The RECONNECT trial is designed to evaluate the efficacy and safety of Zygel (cannabidiol formulated in a transdermal gel) in children and adolescents with Fragile X syndrome (FXS).
  • If successful, we believe the study could serve as the basis for Zygel approval in the U.S. for patients with FXS.
  • Potentially having an approved treatment option for these vulnerable patients would be a significant step forward for FXS patients and their families.
  • Zynerba Pharmaceuticals is the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders.