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NEWS

Denali Therapeutics Announces Publishing of P-I/II Study of Tividenofusp Alfa (DNL310) in NEJM for Hunter Syndrome (MPS II)

Shots: The NEJM published results from the P-I/II study of Denali Therapeutics’ tividenofusp alfa (DNL310) for the treatment of Hunter syndrome (MPS II) The study evaluated treatment in 47 ERT-naïve (n=15) and previously treated (n=32) study participants (aged 0.3–13 [median, 5] years) demonstrating reduced levels of mean CSF levels of HS, mean urine HS levels,…
January 2, 2026

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NEWS

Chiesi Group Collaborates with Aliada Therapeutics to Develop Enzyme Replacement Therapies for Lysosomal Storage Disorders

Shots: Chiesi Group has entered into an exclusive license deal with Aliada Therapeutics, an AbbVie subsidiary, to advance its Blood-Brain Barrier (BBB)-crossing platform technology for developing therapies targeting lysosomal storage disorders As per the deal, Chiesi will receive an exclusive global license to develop & commercialize enzyme replacement therapies using its BBB-crossing platform, with Aliada receiving…
November 13, 2025

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VIEWPOINTS_Angela Cacace_2024
VIEWPOINTS

Transformative Neurodegenerative Disorder Care: Angela Cacace in Conversation with PharmaShots

Shots: Angela Cacace Sr. VP, of Neuroscience and Platform Biology at Arvinas, shares insights from the preclinical trials evaluating ARV-102 for treatment of neurodegenerative diseases and dosing people with the first oral formulation (ARV-102) to treat neurodegenerative diseases ARV-102, a novel oral PROTAC® protein degrader, is designed to cross the blood-brain barrier and target leucine-rich repeat kinase…
April 29, 2024

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