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Featured ArticleNovember 27, 2023Important new ALS insights
About 5,000 people are diagnosed with ALS each year in the United States, and most people with ALS live for only two to five years after diagnosis. -
eNewsOctober 30, 2023Speeding the pace of rare disease research
The Rare Disease Translational center is driving the rare disease research pipeline. -
Research HighlightSeptember 25, 2023Immunometabolism research unearths a potential treatment for lupus
Chih-Hao “Lucas” Chang's immune system and metabolism research have led him to a prospective treatment option for the autoimmune disease systemic lupus erythematosus (SLE or lupus). -
Featured ArticleJune 16, 2023The rare disease odyssey of the Panwala family
Parents of a child with INAD, a rare nervous-system disorder, partner with the JAX Rare Disease Translational Center to test therapies.
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Featured ArticleNovember 17, 2023Spreading ALS awareness and changing patient outcomes
Members of the RDTC and Bar Harbor Preclinical Services participated in a walk to raise awareness for ALS. -
Press ReleaseOctober 30, 2023Highlights and Takeaways from the 2023 NORD Summit
Representatives from JAX attended the 2023 National Organization for Rare Disorders Rare Diseases and Orphan Products Breakthrough Summit. -
Featured ArticleAugust 24, 2023New FDA-approved treatment for Friedreich’s ataxia patients
For the first time, there's an FDA approved treatment for Friedreich’s ataxia (FA). This is a major step forward for the FA community, but is something even better on the horizon? -
Research HighlightJune 02, 2023Bringing preclinical genome editing to the clinic
Supported by an NIH grant, Cat Lutz will lead a multi-institutional team to develop and validate new gene editing-based therapeutic approaches for four neurological conditions lacking effective treatments.
Education Resources
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