- Clinical trials showed that surfactant—a substance that keeps air sacs in the lungs inflated—helps premature infants breathe. As a result, the lives of thousands of babies who would otherwise die of respiratory failure are saved each year.
- Clinical trials showed that giving antiretroviral therapy (AZT) to pregnant women with HIV-AIDS prevents HIV transmission. We have saved the lives of thousands of children born in this country every year thanks to this research.
- As a result of repeated clinical trials in children with cancer, most children who develop leukemia survive. Only 50 years ago, acute leukemia was almost universally fatal in young children.
- Prior to the development of the polio vaccine, 15,000 American children developed paralytic polio each year. In the last five years, there have been no cases of paralytic polio in the United States. This advance was only possible through clinical trials in children.
Articles which demonstrate why research is important
Gene linked to intellectual ability affects memory replay in mice
- Press Release
- Published: June 5, 2018
Researchers at the RIKEN Center for Brain Science in Japan have discovered that a gene associated with human intellectual ability is necessary for normal memory formation in mice. Published in Nature Neuroscience, the study shows that mice with only one copy of the gene replay shorter fragments of their previous experiences during periods of rest, impairing their ability to consolidate memories.Read Full Article
Models for discovery of targeted therapy in genetic epileptic encephalopathies
- By Snezana Maljevic, Christopher A. Reid, Steven Petrou
- First published: July 25, 2017
Epileptic encephalopathies are severe disorders emerging inthefirst days to years of life that commonly includerefractory seizures, various types of movement disorders,and different levels of developmental delay. In recent years,manyde novooccurring variants have been identified inindividuals with these devastating disorders.Read Full Article
FDA Approves Spinraza for SMA
- By Cure SMA
- Published: January 5, 2017
On December 23, the FDA announced that it has approved SpinrazaTM (nusinersen) to treat spinal muscular atrophy, making it the first-ever FDA-approved therapy for SMA. We are thrilled to see our community’s efforts culminate in the approval of Spinraza: not only the first-ever approved treatment for this disease, but also one that addresses the underlying genetic cause of SMA.Read Full Article