Patricia A. Fraser

Phase 1/2 trial of the HPK1 inhibitor NDI-101150 as monotherapy and in combination with pembrolizumab: Clinical update.

Marcus Smith Noel, Kurt C. Demel, Bhaskar Srivastava, Scott R. Daigle et autres

3083 Background: NDI-101150 is a potent, selective, oral inhibitor of hematopoietic progenitor kinase 1 (HPK1), with a different immunotherapy mechanism to other checkpoint inhibitors. NDI-101150 reactivates anti-tumor activity of T-cells, B-cells and dendritic cells (DCs), even under immunosuppressive conditions. Methods: Safety and …

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Choosing to End African American Health Disparities in Patients With Systemic Lupus Erythematosus

Matthew H. Liang, Edward R. Lew, Patricia A. Fraser, Cindy Flower et autres

Systemic lupus erythematosus (SLE) is three times more common and its manifestations are more severe in African American women compared to women of other races. It is not clear whether this is due to genetic differences or factors related to the physical …

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751 Monotherapy results from an ongoing phase 1a dose escalation study of NDI-101150, a highly selective oral hematopoietic progenitor kinase 1 (HPK1) inhibitor

David Sommerhalder, Marcus Smith Noel, Scott Boiko, Scott R. Daigle et autres

Background NDI-101150 is a novel, oral, highly selective small molecule inhibitor of HPK1, a MAP4K family kinase that negatively regulates T cells, B cells, and dendritic cells. HPK1 inhibition is postulated to enhance immune responses and exert anti-tumor activity as a single …

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One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency

George A. Díaz, Simon Allan Jones, Maurizio Scarpa, Eugen Mengel et autres

Correction to: Genetics in Medicine 2022; https://doi.org/10.1038/s41436-021-01156-3In the article “One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency” by Diaz GA et al (Genet Med 2021;23:1543-1550), there were two typographical errors. In …

One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency

George A. Díaz, Simon Allan Jones, Maurizio Scarpa, Eugen Mengel et autres

Purpose To assess olipudase alfa enzyme replacement therapy for non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in children. Methods This phase 1/2, international, multicenter, open-label trial (ASCEND-Peds/NCT02292654) administered intravenous olipudase alfa every 2 weeks with intrapatient dose escalation to 3 …

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Children treated with olipudase alfa for chronic acid sphingomyelinase deficiency show meaningful improvement on clinically relevant outcomes and an overall favorable safety profile: 1-year results of the ASCEND-Peds trial

George A. Díaz, Roberto Giugliani, Nathalie Guffon, Simon A. Jones et autres

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Adults with chronic acid sphingomyelinase deficiency show significant visceral, pulmonary, and hematologic improvements after enzyme replacement therapy with olipudase-alfa: 1-year results of the ASCEND placebo-controlled trial

Melissa Wasserstein, Laila Arash‐Kaps, Antonio Barbato, Renata C. Gallagher et autres

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P.69NEO1 and NEO-EXT studies: exploratory efficacy of repeat avalglucosidase alfa dosing for up to 5 years in participants with late-onset Pompe disease (LOPD)

Benedikt G. H. Schoser, Richard J. Barohn, Barry J. Byrne, Özlem Göker-Alpan et autres

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Pesticide exposure and risk of systemic lupus erythematosus in an urban population of predominantly African-American women

Jessica N. Williams, Corine Sinnette, Susan Malspeis, Christine Gibson Parks et autres

OBJECTIVE: Past studies have reported associations between pesticide exposure and the risk of systemic lupus erythematosus (SLE). Residential pesticide exposure has been less well studied than agricultural exposure. The purpose of this study was to assess SLE risk associated with residential pesticide …

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Efficacy and Safety of Atacicept in Patients With Systemic Lupus Erythematosus

Joan T. Merrill, Daniel J. Wallace, Stephen D. Wax, Amy H. Kao et autres

OBJECTIVE: To evaluate the efficacy and safety of atacicept, an antagonist of B lymphocyte stimulator/APRIL-mediated B cell activation, in patients with systemic lupus erythematosus (SLE). METHODS: ADDRESS II is a 24-week, multicenter, randomized, double-blind, placebo-controlled, parallel-arm, phase IIb study evaluating the safety …

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8 Efficacy and safety of atacicept in patients with systemic lupus erythematosus: results of a 24-week randomisedrandomized, placebo-controlled, phase iib study

JT Merrill, DJ Wallace, Stephen D. Wax, Amy H. Kao et autres

Background and aims Atacicept targets B-cell stimulating factors, BLyS and APRIL. ADDRESS II was a phase IIb, multicenter study (NCT01972568) investigating the efficacy and safety of atacicept in SLE. Methods Patients with active (SLEDAI-2K≥6), autoantibody-positive SLE receiving standard therapy were randomised to …

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Designing an intervention for women with systemic lupus erythematosus from medically underserved areas to improve care: a qualitative study

CH Feldman, BL Bermas, Melanie J. Zibit, Patricia A. Fraser et autres

OBJECTIVE: Systemic lupus erythematosus (lupus) disproportionately affects women, racial/ethnic minorities and low-income populations. We held focus groups for women from medically underserved communities to discuss interventions to improve care. METHODS: From our Lupus Registry, we invited 282 women, ≥18 years, residing in …

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