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Your search keyword '"David Kronn"' showing total 7 results
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7 results on '"David Kronn"'

1. Higher dose alglucosidase alfa is associated with improved overall survival in infantile-onset Pompe disease (IOPD): data from the Pompe Registry

Author

Priya S. Kishnani, David Kronn, Shugo Suwazono, Alexander Broomfield, Juan Llerena, Zuhair Nasser Al-Hassnan, Julie L. Batista, Kathryn M. Wilson, Magali Periquet, Nadia Daba, Andreas Hahn, and Yin-Hsiu Chien

Subjects
Alglucosidase alfa, Dose, Enzyme replacement therapy, Infantile onset Pompe disease, Pompe disease, Pompe registry, Medicine
Abstract

Abstract Background Studies indicate that doses of alglucosidase alfa (ALGLU) higher than label dose (20 mg/kg every other week) improve clinical outcomes in infantile-onset Pompe disease (IOPD). We investigated data from the Pompe Registry to determine the association between ALGLU dose and survival in IOPD. Results We included 332 IOPD patients from the Registry as of January 2022 who had cardiomyopathy and were first treated at age 0 to 4 times label dose), current dose, and lagged dose. 81% patients received label dose at treatment initiation. Over time, 52% received a higher dose. Higher ALGLU dose over time was associated with improved survival: adjusted HR 0.40 (95% CI 0.22–0.73, p = 0.003) per 1-unit increase in average relative dose, with similar results for invasive ventilation-free survival (adjusted HR 0.48, 95% CI 0.28–0.84; p = 0.010). The association was consistent in patients first treated before or after 3 months of age and did not vary significantly by CRIM status. Results for current and lagged dose were similar to average dose. Conclusions Higher ALGLU doses were associated with significantly improved overall and invasive ventilator-free survival in IOPD. Results were consistent across sensitivity analyses.

Published
2023
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3. Motor Responses in Pediatric Pompe Disease in the ADVANCE Participant Cohort

Author

Tina, Duong, Priya S, Kishnani, Kristina, An Haack, Meredith C, Foster, James B, Gibson, Catherine, Wilson, Si Houn, Hahn, Richard, Hillman, David, Kronn, Nancy D, Leslie, Loren D M, Peña, Susan E, Sparks, David W, Stockton, Pranoot, Tanpaiboon, and John W, Day

Subjects
Cohort Studies, Neurology, Glycogen Storage Disease Type II, Motor Skills, Humans, Enzyme Replacement Therapy, Neurology (clinical), Child
Abstract

Background: ADVANCE (NCT01526785) presented an opportunity to obtain a more nuanced understanding of motor function changes in treatment-experienced children with Pompe disease receiving 4000L-production-scale alglucosidase alfa for 52 weeks. Objective: To estimate minimal detectable change (MDC) and effect size on Gross Motor Function Measure-88 (GMFM-88) after 52 weeks of 4000L alglucosidase alfa (complete data N = 90). Methods: The GMFM-88 mean total % score changes, MDC, and effect size were analyzed post hoc by Pompe Motor Function Level at enrollment, age groups at enrollment, and fraction of life on pre-study 160L-production-scale alglucosidase alfa. Results: Overall, participants aged

Published
2022

4. Safety and efficacy of avalglucosidase alfa in individuals with infantile-onset Pompe disease enrolled in the phase 2, open-label Mini-COMET study: The 6-month primary analysis report

Author

Priya S. Kishnani, David Kronn, Anaïs Brassier, Alexander Broomfield, James Davison, Si Houn Hahn, Satoko Kumada, François Labarthe, Hirotaka Ohki, Samia Pichard, S. Grace Prakalapakorn, Kristina An Haack, Barbara Kittner, Xianzhang Meng, Susan Sparks, Catherine Wilson, Atef Zaher, and Yin-Hsiu Chien

Subjects
Genetics (clinical)
Abstract

Mini-COMET (NCT03019406; Sanofi) is a phase 2, open-label, ascending-dose, 3-cohort study, evaluating avalglucosidase alfa safety, pharmacokinetics, and efficacy in individuals with infantile-onset Pompe disease aged18 years who previously received alglucosidase alfa and showed clinical decline (cohorts 1 and 2) or suboptimal response (cohort 3).During a 25-week primary analysis period, cohorts 1 and 2 received avalglucosidase alfa 20 and 40 mg/kg every other week, respectively, for 6 months, whereas cohort 3 individuals were randomized (1:1) to receive avalglucosidase alfa 40 mg/kg every other week or alglucosidase alfa (current stable dose) for 6 months.In total, 22 individuals were enrolled (cohort 1 [n = 6], cohort 2 [n = 5], cohort 3-avalglucosidase alfa [n = 5], and cohort 3-alglucosidase alfa [n = 6]). Median treatment compliance was 100%. None of the individuals discontinued treatment or died. Percentages of individuals with treatment-emergent adverse events were similar across dose and treatment groups. No serious or severe treatment-related treatment-emergent adverse events occurred. Trends for better motor function from baseline to week 25 were observed for 40 mg/kg every other week avalglucosidase alfa compared with either 20 mg/kg every other week avalglucosidase alfa or alglucosidase alfa up to 40 mg/kg weekly.These data support the positive clinical effect of avalglucosidase alfa in patients with infantile-onset Pompe disease previously declining on alglucosidase alfa.

Published
2023

5. Mini-COMET study: Effects of 97 weeks of avalglucosidase alfa dosing on ptosis in participants with infantile-onset Pompe disease who were previously treated with alglucosidase alfa

Author

Priya S. Kishnani, Marguerite C. Weinert, James Davison, Alexander Broomfield, Yin-Hsiu Chien, Sihoun Hahn, David Kronn, Satoko Kumada, Hirotaka Ohki, Samia Pichard, Kristina An Haack, Swathi Tammireddy, Tianyue Zhou, and Sasapin G. Prakalapakorn

Subjects
Endocrinology, Endocrinology, Diabetes and Metabolism, Genetics, Molecular Biology, Biochemistry
Published
2023

6. OP016: Mini-COMET: Safety and efficacy of ≥97 weeks’ avalglucosidase alfa in infantile-onset Pompe disease participants previously treated with alglucosidase alfa

Author

David Kronn, James Davison, Anaïs Brassier, Alexander Broomfield, Si Houn Hahn, Satoko Kumada, François Labarthe, Hirotaka Ohki, S. Grace Prakalapakorn, Kristina An Haack, Xianzhang Meng, Susan Sparks, Swathi Tammireddy, Catherine Wilson, Atef Zaher, Tianyue Zhou, Yin-Hsiu Chien, Priya Kishnani, and null On behalf of the Mini-COMET investigators

Subjects
Genetics (clinical)
Published
2022

7. Mini-COMET study: Safety, biomarker, and efficacy data after avalglucosidase alfa dosing for ≥ 97 weeks in participants with infantile-onset pompe disease (IOPD) previously treated with alglucosidase alfa who had demonstrated clinical decline

Author

David Kronn, James Davison, Anaïs Brassier, Alexander Broomfield, Si Houn Hahn, Satoko Kumada, François Labarthe, Hirotaka Ohki, S. Grace Prakalapakorn, Kristina An Haack, Xianzhang Meng, Susan Sparks, Swathi Tammireddy, Catherine Wilson, Atef Zaher, Tianyue Zhou, Yin-Hsiu Chien, and Priya S. Kishnani

Subjects
Endocrinology, Endocrinology, Diabetes and Metabolism, Genetics, Molecular Biology, Biochemistry
Published
2022

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