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News Release
NIH researchers have identified a treatment that significantly decreases the risk of stroke in children with a rare genetic disease called Deficiency of Adenosine Deaminase Type 2 (DADA2).
… reason. … Benefits of the Treatment … Anti-inflammatory drug halts strokes in children with rare disease … NIH …
News Release
NHGRI researchers have identified new genes associated with the Erdheim-Chester disease (ECD) and some possible new therapies.
… and protect the body from infection. ECD has no standard therapy, although consensus guidelines for clinical … the most common treatment for ECD has been interferon , a drug that interferes with the division of cancer cells and …
Research at NHGRI
The Metabolic Medicine Branch houses clinical and laboratory research programs that focus on the development of new therapies for patients with genetic disorders.
… and their research teams develop gene addition therapy and gene editing approaches to treat metabolic … of single cells and explore the side-effects of gene therapy. The MMB is invested in mentorship and supports …
Staff
Dr. Acosta is a board-certified pediatric neurologist with the NIH Undiagnosed Diseases Program's GM1 gene therapy trial.
… the NIH Undiagnosed Disease Program (UDP) and the GM1 gene therapy program led by Dr. Cynthia Tifft. … Her main area of … A, Tinoco D, Vance M, Yavorsky C. Methods for Neuroscience Drug Development: Guidance on Standardization of the Process … V. Clinician- and Patient-reported Endpoints in CNS Orphan Drug Clinical Trials: ISCTM Position Paper on Best Practices …
News Release
NHGRI researchers have released new educational materials to help the sickle cell disease community learn about gene therapies for the disease.
… The Democratizing Education for Sickle Cell Disease Gene Therapy Project, led by Vence Bonham Jr., J.D., NHGRI acting … disease and their support networks to learn about the gene therapy clinical trial process, its benefits and risks, … community information about what participating in a gene therapy clinical trial entails,” said Kiana Amini, a …
News Release
NIH grants aim to gauge if genomics can be used to help diagnose diabetes, manage heart disease and guide cancer clinicians to the most effective drugs.
… which studies how a patient's genetic make-up affects drug responses. A third grant examines the role of genomic … information into electronic health records can guide drug prescribing and treatment decisions, and ultimately … minority patients. The study will examine specific drug-gene associations in the three cardiovascular drugs and …
News Release
Investigators with The Cancer Genome Atlas have discovered genomic differences in head and neck cancers caused by infection with the human papillomavirus.
… new smoking-related cancer subtypes and potential new drug targets, and found numerous genomic similarities with … of the National Institutes of Health. The U.S. Food and Drug Administration-approved HPV vaccines should be able to … The investigators also discovered new clues about drug resistance in head and neck cancers. They found that …
News Release
NIH awards more than $64 million to for a database of human cellular responses called the Library of Integrated Network-based Cellular Signatures or LINCS.
… you could figure out patterns of toxicity of potential new drug compounds by looking at cellular responses and finding … responses to drugs was more accurate than conventional drug-potency tests. This recognition may point the way toward … a rational approach to drug discovery and personalizing therapy. Oregon Health and Science University (OHSU), …
For Patients and Families
Neglected diseases are conditions that inflict severe health burdens on the world's poorest people.
… on the world's poorest people and are often overlooked by drug developers or by others instrumental in drug access. … Neglected diseases are conditions that inflict … are said to be neglected if they are often overlooked by drug developers or by others instrumental in drug access, …
News Release
The U.S. Food and Drug Administration has approved the first treatment for progeria, a rare and fatal pediatric disease, characterized by dramatic, rapid aging beginning in childhood.
… rare, fatal pediatric disease. … What The U.S. Food and Drug Administration has approved the first treatment for … of a mouse model, and the demonstration that this drug class could provide benefit. Progeria, also known as … FDA-approved treatment for progeria … The U.S. Food and Drug Administration has approved the first treatment for …