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News Release
Doctors and researchers reunite with patient who received gene therapy for GM1 gangliosidosis.
… Fewer than 5% of rare disorders have effective treatments. GM1 gangliosidosis is amongst the majority for … gene therapy can provide long-term benefit.  Like all treatments, gene therapy has risks. Sometimes the body’s …
Clinical Research
The Attention Deficit/Hyperactivity Disorder (ADHD) Genetic Research Study explores the genetic factors contributing to ADHD to improve treatment in the future.
… the diagnosis of ADHD and the development of improved treatments, possibly including individually tailored treatments. Discovering the genes that are related to ADHD …
Media Availability
The Cancer Genome Atlas identified molecular subtypes to help clinicians determine which tumors are more aggressive and which will respond to treatment.
… aggressive and which are more likely to respond to certain treatments. Their findings confirmed that PTCs are driven … medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For …
Clinical Research
NHGRI researchers work with patients and families to explore how genes cause or influence diseases, and develop more effective diagnostics and treatments.
… and develop new and more effective diagnostics and treatments. … The MINI section aims to define the … disorders, and to develop new and more effective tests and treatments. Deciding whether to participate in a clinical … researchers develop new diagnostic tests, more effective treatments, and better ways of managing diseases with genetic …
News Release
Knowing your family health history can aid in detecting unique disease risks and lead to better prevention and treatment.
News Release
A genetic mutation that triggers bladder cancer in dogs is identical to a mutation that is implicated in multiple human cancers.
Research at NHGRI
NHGRI researchers work with patients and families to understand of how genes influence disease and develop more effective diagnostics and treatments.
… and develop new and more effective diagnostics and treatments. … Current Clinical Studies … Clinical Research … … diseases and develop more effective diagnostics and treatments.​ ​ … Clinical Research, Genetic Disorders, … disease and develop more effective diagnostics and treatments. … Research at NHGRI …
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).
News Release
NHGRI researchers have identified a genomic signature for some aggressive prostate tumors, which may help healthcare providers pinpoint treatment options.
Event
A joint NHGRI-NCATS hosted meeting bringing together researchers, clinicians, families and patient advocates from around the world to discuss the natural history, disease pathophysiology, genetics and treatments for rare inborn metabolic errors.
… the natural history, disease pathophysiology, genetics and treatments for rare inborn metabolic errors. We will also … the natural history, disease pathophysiology, genetics and treatments for rare inborn metabolic errors. … A joint … the natural history, disease pathophysiology, genetics and treatments for rare inborn metabolic errors. … Event …