During June, Neurogene will participate in the Goldman Sachs 45th Annual Global Healthcare Conference, the 2024 IRSF (International Rett Syndrome Foundation) Rett Syndrome Scientific Meeting, and the TD Cowen Genetic Medicines & RNA Summit. Learn more here: https://bit.ly/4bJU3Qh
Neurogene Inc.’s Post
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Clinical trials for genetic therapies may pose risks and benefits that are different from other CF clinical trials, and asking specific questions can help you understand whether a trial is right for you. Download our guide, “Questions to Ask When Enrolling in a Genetic Therapy Clinical Trial,” to help navigate conversations with your research team. https://lnkd.in/eJZZmQbg
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Complement system dysregulation can contribute to #kidneydiseases such as IgA nephropathy. Watch this video to learn about laboratory methods for evaluation of the complement system, including biomarker, autoantibody, and genetic tests.
HCPs: Watch This Video To Learn More About Complement Testing
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Spinocerebellar ataxia type 3 (SCA3) and type 1 (SCA1) are both genetic disorders falling under the category of spinocerebellar ataxias (SCAs), a group of progressive, inherited conditions characterized by damage to the cerebellum and other parts of the nervous system. Antisense oligonucleotide (ASO) RNA modulating therapies are among the approaches being explored for their potential to address the genetic abnormalities associated with these conditions. Discover more: https://vicotx.com/ #PrecisionChemistry #SCA3 #SCA1
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When should you consider chromosomal microarray testing? Learn about our testing, which is supported by the experience and expertise of our laboratory directors and genetic counselors to ensure we provide clinically actionable test results and interpretations. https://bit.ly/3vKlIAF
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At 4DMT, we are advancing enhanced therapeutic vectors with the potential to transform treatment of people with genetic diseases like #CysticFibrosis (CF). 4D-710 is our investigational genetic medicine candidate for the treatment of CF and is currently being evaluated in the AEROW clinical trial. Learn more about 4D-710.
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If you have been diagnosed with Lynch syndrome or a BRCA1/2 mutation, and have family members who have not had genetic testing, consider enrolling in the IGNITE-TX trial. Read more here: https://ow.ly/V1xn50QtLcy.
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A great presentation at 2023 MDA conference by Dr. Peter Marks on the future and promise of gene therapies and CBER's efforts and commitment to move these forward through the regulatory framework: https://lnkd.in/g-e8dx9N.
2023 MDA Conference: FDA Keynote Address On Genetic Therapy, Peter Marks MD PhD
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🔬 Discover gene-diseases associations and genetic testing recommendations in vascular anomalies with this week's #researchhighlight! Our vascular anomalies working group's latest study emphasizes the growing importance of genetic testing for somatic variants, offering valuable insights. Collaborating with clinicians and laboratory specialists from 11 European centers, they reviewed genes linked to non-hereditary vascular malformations. The study identified 24 core genes and 45 gene-phenotype associations, providing comprehensive genetic testing recommendations. This pioneering research improves data interpretation, elevates clinical diagnostics, and paves the way for improved patient care and treatment. Find out more about this study: https://lnkd.in/ejz4TJes #vascularanomalies #rarediseases #HealthcareResearch #BetterOutcomes #genetictesting #diagnosticprocess
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We caught up with Dr Tim Lee, the lead consultant for the Leeds Children's Cystic Fibrosis Centre, to learn more about how he started his career in CF, his interest in genetic therapies, and his useful barbeque tip! Click the link to read Tim's blog: https://lnkd.in/eyPyX56p To find out more about CF genetic therapy trials, check out our genetic therapy resources. #cfnews #genetictherapies #cysticfibrosis
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Download the free GBinsight Case Study Booklet: https://lnkd.in/ggMZZXq2 Case Study 12 (Page 23): Causes of hypertriglyceridemia beyond FCS and FPLD: The GBinsight Dyslipidemia and ASCVD Comprehensive genetic analysis identified one pathogenic/likely pathogenic variant in the CREB3L3 gene and two possible large effect size variants in the APOE and KSR2 genes. The polygenic risk analysis identified high-risk scores for Hypertriglyceridemia.
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