Punam Malik, MD

Safety and Efficacy of Aru-1801 in Patients with Sickle Cell Disease: Early Results from the Phase 1/2 Momentum Study of a Modified Gamma Globin Gene Therapy and Reduced Intensity Conditioning. Grimley, M; Asnani, M; Shrestha, A; Felker, S; Lutzko, C; Arumugam, PI; Witting, S; Knight-Madden, J; Niss, O; Quinn, CT; et al. Blood. 2021; 138:3970-3970.

Towards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI). Adair, JE; Androski, L; Bayigga, L; Bazira, D; Brandon, E; Dee, L; Deeks, S; Draz, M; Dubé, K; Dybul, M; et al. Gene Therapy. 2021.

Rapid and automated quantitation of dense red blood cells: A robust biomarker of hydroxyurea treatment response. Sadaf, A; Quinn, CT; Korpik, JB; Pfeiffer, A; Reynaud, M; Niss, O; Malik, P; Ware, RE; Kalfa, TA; McGann, PT. Blood Cells, Molecules, and Diseases. 2021; 90.

Early initiation of hydroxyurea (hydroxycarbamide) using individualised, pharmacokinetics-guided dosing can produce sustained and nearly pancellular expression of fetal haemoglobin in children with sickle cell anaemia. Quinn, CT; Niss, O; Dong, M; Pfeiffer, A; Korpik, J; Reynaud, M; Bonar, H; Kalfa, TA; Smart, LR; Malik, P; et al. British Journal of Haematology. 2021; 194:617-625.

Cardiac pathophysiology in sickle cell disease. Gbotosho, OT; Taylor, M; Malik, P. Journal of Thrombosis and Thrombolysis. 2021; 52:248-259.

Implementation of near-universal hydroxyurea uptake among children with sickle cell anemia: A single-center experience. Karkoska, K; Todd, K; Niss, O; Clapp, K; Fenchel, L; Kalfa, TA; Malik, P; Quinn, CT; Ware, RE; McGann, PT. Pediatric Blood and Cancer. 2021; 68.

FT-4202, an oral PKR activator, has potent antisickling effects and improves RBC survival and Hb levels in SCA mice. Shrestha, A; Chi, M; Wagner, K; Malik, A; Korpik, J; Drake, A; Fulzele, K; Guichard, S; Malik, P. Blood Advances. 2021; 5:2385-2390.

Safe and Effective In Vivo Targeting and Gene Editing in Hematopoietic Stem Cells: Strategies for Accelerating Development. Cannon, P; Asokan, A; Czechowicz, A; Hammond, P; Kohn, DB; Lieber, A; Malik, P; Marks, P; Porteus, M; Verhoeyen, E; et al. Human Gene Therapy. 2021; 32:31-42.

Left atrial dysfunction in sickle cell anemia is associated with diffuse myocardial fibrosis, increased right ventricular pressure and reduced exercise capacity. Alsaied, T; Niss, O; Tretter, JT; Powell, AW; Chin, C; Fleck, RJ; Cnota, JF; Malik, P; Quinn, CT; Nagueh, SM; et al. Scientific Reports. 2020; 10.

Progression of albuminuria in patients with sickle cell anemia: a multicenter, longitudinal study. Niss, O; Lane, A; Asnani, MR; Yee, ME; Raj, A; Creary, S; Fitzhugh, C; Bodas, P; Saraf, SL; Sarnaik, S; et al. Blood Advances. 2020; 4:1501-1511.