This rare disease destroys a child's ability to walk and speak. Could a new drug help? [MSNBC.com]
Denali Therapeutics Inc. (DNLI)
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Source: MSNBC.com
Like other children with Hunter syndrome, Cole is deficient in an enzyme required to break down certain molecules. Over time, toxins accumulate, and the genetic disorder ravages children’s organs, including their heart — and in many cases, their brain, leading to dementia-like symptoms. The condition, also called mucopolysaccharidosis type II, or MPS II, affects about 500 people in the U.S., nearly all of them boys.Experts believe the newly approved drug, an intravenous enzyme replacement therapy manufactured by Denali Therapeutics, will be a game changer — especially because the current standard of care slows only the physical aspects of the illness. Denali’s drug also targets cognitive decline.The new drug will not reverse regressions that have already occurred. But it could extend children’s lives and prevent many symptoms from showing up for those who receive it early.Dr. Joseph Muenzer, an expert in Hunter syndrome and related rare diseases.Allison M. Donnelly for NBC News“If we
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News
- Denali Therapeutics Regains Full Rights to Investigational Therapy DNL593 (PTV:PGRN) for GRN-related Frontotemporal Dementia (FTD-GRN) [Yahoo! Finance]Yahoo! Finance
- Denali Therapeutics Regains Full Rights to Investigational Therapy DNL593 (PTV:PGRN) for GRN-related Frontotemporal Dementia (FTD-GRN)GlobeNewswire
- They're in a race to save their children with a rare disease. A newly approved drug has given them hope. [MSNBC.com]MSNBC.com
- The $578 Billion Regenerative Medicine Market Has a Manufacturing Problem. One Company Just Solved the First StepPR Newswire
- Denali Therapeutics (DNLI) Is Down 13.9% After First BBB-Crossing Hunter Therapy Wins FDA Nod - What's Changed [Yahoo! Finance]Yahoo! Finance
DNLI
Earnings
- 2/26/26 - Beat
DNLI
Sec Filings
- 4/3/26 - Form 8-K
- 3/31/26 - Form 8-K
- 3/26/26 - Form SCHEDULE
- DNLI's page on the SEC website