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New insights into the mechanisms of "Niemann-Pick type C" in children
December 06, 2024
“We typically associate dementia with elderly people. However, there are also dementias that manifest in children and lead to death already by the age of 30 or even earlier, such as Niemann-Pick type C.”
Dr. Sabina Tahirovic, neuroscientist at DZNE’s Munich site
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WNDU
Notre Dame celebrates first FDA-approved treatment for Niemann-Pick Type C disease
October 17, 2024
The University of Notre Dame is celebrating a big medical breakthrough that’s been decades in the making. Within the past month, the FDA has announced the approval of new drugs to treat the rare disease Niemann-Pick Type C. You may remember, that’s the same disease that claimed the lives of three grandchildren of former Notre Dame football coach Ara Parseghian.
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After landmark rare disease approval, IntraBio ready to 'hit the ground running' with Aqneursa launch
September 30, 2024
With its first approval in hand, privately held IntraBio is embarking upon the difficult task of marketing a drug for an ultra-rare disease. Still, with months of prep work and outreach under its belt, the company is already proving it can hit the ground running.