IntraBio, co-founded by Department of Pharmacology academics Professors Fran Platt, Antony Galione and Grant Churchill, ran a successful international clinical trial for a new drug Aqneursa which showed significantly improved neurological outcomes over a 12 week period for a cohort of 60 patients, aged four years and over, with a confirmed diagnosis of NPC compared to a control group. Despite the recent proliferation of Drug Discovery Units in many Universities, it is nevertheless still rare for academics to take a new molecular entity from pre-clinical to phase III clinical trials culminating in regulatory approval and marketing for patients. The first prescriptions for Aqneursa have already been written.
NPC is a rare genetic disease that results in progressive neurological symptoms and organ dysfunction. It is caused by changes in either the NPC1 or NPC2 gene, affecting the transport of cholesterol and other lipids out of the lysosome. As a result, cells do not function as they should, ultimately causing organ damage. On average, individuals affected by this devastating disease only live for about 13 years. The new drug, Aqneursa, is only the third drug approved for the treatment of NPC, including miglustat, which was the first drug to be approved for lysosomal storage diseases that was developed by the Platt laboratory.
Read the full story on the Department of Pharmacology website.
more recommended stories
Pink Noise Boosts Brain Waste Clearance During SleepKey Points Pink noise delivered during.
Psilocybin Microdosing Research Raises Key QuestionsKey points: Psilocybin microdosing research shows.
Maternal Illness Linked to Epigenetic Fetal Brain ChangesMaternal illness during pregnancy may influence.
SGLT2 Inhibitors Boost Cardiac Energy Through PANK1Key Points SGLT2 inhibitors directly activate.
Daraxonrasib Approved for Metastatic Pancreatic CancerKey Points FDA approved daraxonrasib (RASONQUE).
Nicotine E-Cigarettes Help More Smokers Quit Than NRTKey Points Nicotine e-cigarettes helped more.
AI Identifies Key Gene Driving Hematopoietic Stem Cell AgingKey Points AI analysis identified Pbx1.
Intravenous Iron Improves Recovery After Heart SurgeryKey points A large multicenter randomized.
Childhood Epilepsy Linked to Lower Educational AchievementKey Takeaways Childhood epilepsy was associated.
Laser Treatment for Glioblastoma Shows Survival BenefitKey Points A multicenter analysis of.
Leave a Comment