V. Karsten

发表

Phase 3 Multicenter Study of Revusiran in Patients with Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Cardiomyopathy (ENDEAVOUR)

R. Falk, D. Judge, M. Maurer, 2020, Cardiovascular Drugs and Therapy.

DISCOVERY: prevalence of transthyretin (TTR) mutations in a US-centric patient population suspected of having cardiac amyloidosis

M. Maurer, H. Taylor, J. Gollob, 2020, Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis.

Effects of Patisiran, an RNA Interference Therapeutic, on Cardiac Parameters in Patients With Hereditary Transthyretin-Mediated Amyloidosis: Analysis of the APOLLO Study

S. Solomon, A. Shah, M. Maurer, 2019, Circulation.

Phase I First-in-Humans Trial of ALN-TTRsc, a Novel RNA Interference Therapeutic for the Treatment of Familial Amyloidotic Cardiomyopathy (FAC)

Tracy S. Zimmermann, J. Gollob, R. Falzone, 2013 .

Retrospective study of a TTR FAP cohort to modify NIS+7 for therapeutic trials

B. Bettencourt, M. Benson, C. Klein, 2014, Journal of the Neurological Sciences.

Phase I Trial of VNP 40101 M ( Cloretazine ) in Childrenwith Recurrent BrainTumors : A Pediatric BrainTumor Consortium Study

I. Pollack, H. Friedman, R. Packer, 2008 .

Phase I Trial of VNP40101M (Cloretazine) in Children with Recurrent Brain Tumors: A Pediatric Brain Tumor Consortium Study

I. Pollack, H. Friedman, R. Packer, 2008, Clinical Cancer Research.

Abstract 67: Phase I Safety, Pharmacokinetic, and Pharmacodynamic Results for ALN-PCS, a Novel RNAi Therapeutic for the Treatment of Hypercholesterolemia

J. Ritter, L. Nechev, K. Fitzgerald, 2012, Arteriosclerosis, Thrombosis, and Vascular Biology.

Serial scanning with technetium pyrophosphate (99mTc-PYP) in advanced ATTR cardiac amyloidosis

B. Bettencourt, M. Maurer, W. Blaner, 2016, Journal of Nuclear Cardiology.

Preclinical and Clinical Studies Employing RNA Interference as a Therapeutic for Respiratory Syncytial Virus (RSV) Infection in the Lung

J. Gollob, R. Meyers, J. Cehelsky, 2013 .

Clinical Proof of Concept for a Novel Hepatocyte-Targeting GalNAc-siRNA Conjugate.

B. Bettencourt, Tracy S. Zimmermann, J. Gollob, 2017, Molecular therapy : the journal of the American Society of Gene Therapy.

Phase I study of temozolomide and laromustine (VNP40101M) in patients with relapsed or refractory leukemia.

W. Tse, A. Advani, S. Gerson, 2010, Clinical lymphoma, myeloma & leukemia.

Phase I Study of Cloretazine (VNP40101M), a Novel Sulfonylhydrazine Alkylating Agent, Combined with Cytarabine in Patients with Refractory Leukemia

M. Sznol, H. Kantarjian, G. Garcia-Manero, 2005, Clinical Cancer Research.

Results of a phase 2b multi-center trial of ALN-RSV01 in respiratory syncytial virus (RSV)-infected lung transplant patients

M. Zamora, J. Gollob, J. Gottlieb, 2012 .

Phase 2, open-label extension (OLE) study of revusiran, an investigational RNAi therapeutic for the treatment of patients with transthyretin cardiac amyloidosis

R. Falk, M. Maurer, P. Hawkins, 2015, Orphanet Journal of Rare Diseases.

ALN-RSV01 for prevention of bronchiolitis obliterans syndrome after respiratory syncytial virus infection in lung transplant recipients.

B. Bettencourt, M. Zamora, J. Gollob, 2016, The Journal of heart and lung transplantation : the official publication of the International Society for Heart Transplantation.

Single‐Dose Pharmacokinetics and Pharmacodynamics of Transthyretin Targeting N‐acetylgalactosamine–Small Interfering Ribonucleic Acid Conjugate, Vutrisiran, in Healthy Subjects

M. Sweetser, H. Attarwala, V. Goel, 2020, Clinical pharmacology and therapeutics.

DISCOVERY: a study examining the prevalence of transthyretin mutations in subjects suspected of having cardiac amyloidosis

M. Maurer, H. Taylor, K. Ananthasubramaniam, 2015, Orphanet Journal of Rare Diseases.

Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial

B. Bettencourt, Svetlana Shulga-Morskaya, J. Gollob, 2014, The Lancet.

Single-agent laromustine, a novel alkylating agent, has significant activity in older patients with previously untreated poor-risk acute myeloid leukemia.

S. Solomon, D. DeAngelo, S. O'brien, 2010, Journal of Clinical Oncology.

Cloretazine (VNP40101M), a novel sulfonylhydrazine alkylating agent, in patients age 60 years or older with previously untreated acute myeloid leukemia.

G. Mufti, H. Kantarjian, F. Ravandi, 2006, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

Association of Patisiran, an RNA Interference Therapeutic, With Regional Left Ventricular Myocardial Strain in Hereditary Transthyretin Amyloidosis: The APOLLO Study.

Giampaolo Merlini, S. Solomon, R. Falk, 2019, JAMA cardiology.

CLORETAZINE™ (VNP40101M) Is Active in Elderly Untreated Poor Risk Patients with Acute Myelogenous Leukemia (AML) or High Risk Myelodysplasia (MDS).

G. Mufti, J. Karp, S. O'brien, 2005 .

Effects of Initial Treatment on Survival among Elderly AML Patients: Findings from the SEER-Medicare Database.

J. Menzin, L. Boulanger, V. Karsten, 2006 .

Results of a phase 2b multi-center, randomized, double-blind, placebo-controlled study of an RNAi therapeutic, ALN-RSV01, in respiratory syncytial virus (RSV)-infected lung transplant patients

M. Zamora, J. Gollob, J. Gottlieb, 2012 .

A phase II study of cloretazine (VNP40101M), a novel sulfonylhydrazine alkylating agent, in patients with very high risk relapsed acute myeloid leukemia.

H. Kantarjian, S. Verstovsek, J. Karp, 2006, Leukemia research.

Phase I Trial of VNP40101M (Cloretazine) in Children with Recurrent Brain Tumors: A Pediatric Brain Tumor Consortium Study

I. Pollack, H. Friedman, R. Packer, 2008, Clinical Cancer Research.

Single-agent laromustine, a novel alkylating agent, has significant activity in older patients with previously untreated poor-risk acute myeloid leukemia.

S. Solomon, D. DeAngelo, S. O'brien, 2010, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

558 CTX130 allogeneic CRISPR-Cas9–engineered chimeric antigen receptor (CAR) T cells in patients with advanced clear cell renal cell carcinoma: Results from the phase 1 COBALT-RCC study

N. Agarwal, M. Hurwitz, B. Tran, 2022, Regular and Young Investigator Award Abstracts.

Clinical Proof of Concept for a Novel Hepatocyte-Targeting GalNAc-siRNA Conjugate.

B. Bettencourt, Tracy S. Zimmermann, J. Gollob, 2017, Molecular therapy : the journal of the American Society of Gene Therapy.