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News Release
Researchers are asking if the precision medicine approach will reduce or eliminate the role that race plays in prescribing drugs and in health care overall.
… the complex interplay of ancestry, health, disease and drug response. … In a May 26 New England Journal of Medicine … prescribing drugs and in health care overall. "We focus on drug therapies because that's where the precision medicine … overall. ​ … Precision medicine, drug prescribing, drug therapy, Vence Bonham, longitudinal drug studies, drug …
News Release
A team from the NIH has surmounted a major obstacle to testing potential drug therapies for a rare, genetic condition called Gaucher disease.
… has surmounted a major obstacle to testing potential drug therapies for a rare, genetic condition called Gaucher … of the disease. Using this model, they have now tested a drug compound that has successfully corrected the … called Gaucher disease. ​ … Gaucher disease, drug therapy for Gaucher disease, Ellen Sidransky Ph.D., GBA gene …
News Release
A repurposed cancer drug appears to reduce the severity of lesions and pain experienced by people with Proteus syndrome
… Jeannine Mjoseth … A repurposed cancer drug appears to reduce the severity of lesions and pain … people with Proteus syndrome, a rare genomic disease. The drug, miransertib, was well tolerated, even in children, … using the drug was very different from its use in cancer therapy. In cancer, the objective is to eliminate all …
News Release
NIH researchers have uncovered a key factor in understanding the elevated cancer risk associated with gene therapy.
… the elevated cancer risk associated with gene therapy. They conducted research on mice with a rare disease … hoping their findings may eventually help improve gene therapy for humans. Researchers at the National Human Genome … into a cell. AAV viruses are uniquely suited for gene therapy applications. But one prior study did find an …
Fact Sheet
Gene therapy for sickle cell disease is available to you through FDA-approved therapies and through clinical trials.
… Gene therapy for sickle cell disease is available to you through … there are a number of clinical trials for sickle cell gene therapy, the journey can be similar across all treatments. … will: Be admitted to the hospital. Receive a chemotherapy drug through an intravenous catheter. This process, called …
Fact Sheet
Understanding gene therapy for sickle cell disease
… the Democratizing Education for Sickle Cell Disease Gene Therapy Project, which is led by NHGRI in partnership with … welcomes your feedback about the sickle cell disease gene therapy resources. Please email your comments or questions to … are meant to promote your general understanding of gene therapy for sickle cell disease. We encourage you to use …
News Release
NIH researchers demonstrated that gene therapy may be the best method for correcting the single faulty gene that causes Niemann-Pick disease, type C1.
… (NIH) researchers have demonstrated in mice that gene therapy may be the best method for correcting the single … that causes Niemann-Pick disease, type C1 (NPC1). The gene therapy involved inserting a functional copy of the NPC1 gene … gene therapy. The effect of gene therapy equaled that of a drug called VTS-270, which has been evaluated in preclinical …
Research at NHGRI
The Democratizing Education for Sickle Cell Disease Gene Therapy project developed patient-focused educational materials for the sickle cell community.
… throughout the body. In December 2023, the U.S. Food and Drug Administration (FDA) approved two gene therapies to … the Democratizing Education for Sickle Cell Disease Gene Therapy Project, which is led by NHGRI in partnership with … individuals living with sickle cell disease and the gene therapy and biomedical research communities.  … This …
News Release
SJS/TEN is a severe drug reactions that affect the skin, and one of the most severe of adverse reactions affecting any system in the body
… Necrolysis (SJS/TEN). SJS/TEN is the severest of adverse drug reactions that affect the skin, and one of the most … organized a workshop , in collaboration with the Food and Drug Administration (FDA) and other NIH institutes, to … factors that predispose patients to severe immune mediated drug reactions like SJS/TEN, as well as studying the …
For Patients and Families
​Pharmacogenomics combines the science of how drugs work, called pharmacology, with the science of the human genome, called genomics.​
… genetic makeup, or genome, to choose the drugs and drug doses that are likely to work best for that particular … genetic makeup, or genome, to choose the drugs and drug doses that are likely to work best for that particular … is the breast cancer drug trastuzumab (Herceptin). This therapy works only for women whose tumors have a particular …