A JAX research team led by Cat Lutz launched a project to develop gene therapy strategies and to test small molecules for...
Rare and Orphan Disease Research Tools and Online Resources
Resources for people who are interested in learning more about rare diseases, clinical trials, and research programs.
Families have banded together from across the globe to make awareness and research for SMARD a reality.
Did you know that almost all of us have the sort of genetic mutations that cause rare genetic diseases?
Using the right mouse model can reveal various biological intricacies and may lead to potential therapeutic treatments.In a recent study,...
JAX distributes well-characterized, preclinical mouse models to accelerate drug discovery for rare and orphan diseases.
We engineer new models and enhance existing models through genetic standardization and characterization to ensure reproducibility of data...
Efficacy studies are performed by JAX on mouse models for these diseases:C57BL10.mdx and D2.mdx for DMD, DyW for CMD1A, and A/J for...
We partner with foundations, pharmaceutical and biotech companies, and other scientists to research treatments for uncommon...
JAX has teamed with Lovelace Biomedical, Exemplar Genetics, Iontox and the University of Pennsylvania to win a new $17M contract with the...
New five-year NIH grant totaling $10.6M funds JAX center to fast-track treatment-focused research for rare genetic...
Genetic diversity should be accounted for in pre-clinical tests, and researchers need to select the right model system to mimic human...
What should healthcare professionals do to incorporate genomic medicine into patient care?Here are the keys to genomic risk...
JAX Professor Robert Burgess and collaborators are developing personalized gene therapy for a Texas child suffering from a neuromuscular...
JAX neuroscientist Cat Lutz, Ph.D., gave a TED-style talk about strides in rare disease research as part of the Laboratory’s JAXtaposition...