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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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VIEWPOINTS_Björn Mellgård_2023
VIEWPOINTS

Björn Mellgård shares insights from the Pivotal Phase III data of TAK-755 for congenital thrombotic thrombocytopenic purpura (cTTP)

Shots:  Björn Mellgård Vice President and Global Program Lead, TTP Program, Takeda, shares insights from the P-III data of TAK-755 for the treatment of cTTP   Björn eloquently shares the highlights of the study design for TAK-755, safety and efficacy outcomes, and goes on to share an update from the P-IIIb continuation trial evaluating TAK-755   With…
October 19, 2023

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VIEWPOINTS_Bettina Cockroft_2023
VIEWPOINTS

Bettina Cockroft, SVP & CMO at Sangamo Therapeutics Shares Insights from the Positive P-I/II Data for Fabry Disease Treatment

Shots: Bettina gave the details of the study design and shared the preliminary results from the P-I/II STAAR clinical study evaluating ST-920. The data were presented in an oral presentation at the 29th Congress of the European Society of Gene & Cell Therapy 2022  She also talked about enzyme replacement therapy (ERT) which is currently used for the…
April 24, 2023

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