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DNLI's Hunter Syndrome Drug Gets Breakthrough Therapy DesignationDenali’s wholly owned program, DNL310 or tividenofusp alfa, is an Enzyme Transport Vehicle-enabled iduronate-2-sulfatase (IDS) replacement therapy in development for MPS II (Hunter syndrome).
Feb. 06, 2025 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (NASDAQ: DNLI), today announced the primary analysis of the Phase 1/2 study in 47 participants with Hunter syndrome (MPS II) in the 24-week ...
The UK regulatory authorities have approved the first ever trial of a revolutionary gene therapy for young children diagnosed with Hunter syndrome ... also known as mucopolysaccharidosis type II (MPS ...
Shire’s attempts to develop a drug for cognitive impairment from the rare inherited disease Hunter Syndrome has failed ... and families living with MPS II.” “We are grateful to the children ...
for Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome and RGX-111 for Mucopolysaccharidosis I (MPS I), also known as Hurler syndrome in the United States and Asia. "RGX-121 and RGX ...
The University of Manchester has announced today a groundbreaking gene therapy partnership to ease the lifelong suffering of people with Hunter syndrome. The University has agreed to a worldwide ...
Regenxbio closes strategic partnership with Nippon Shinyaku for MPS diseases: Rockville, Maryland Wednesday, March 5, 2025, 14:00 Hrs [IST] Regenxbio Inc., a leading clinical-stag ...
Regenxbio (RGNX) announced the closing of its previously announced strategic partnership with Nippon Shinyaku. Under the terms of the ...
DNLI reports a narrower-than-expected loss for the fourth quarter of 2024. Denali remains on track to submit a BLA for ...
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