G. Elfring

发表

Progression-free survival is a surrogate for survival in advanced colorectal cancer.

D. Sargent, M. Buyse, J. Pignon, 2007, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

R. Finkel, E. Bertini, S. Peltz, 2017, The Lancet.

Multiple-stage procedures for drug screening.

G. Elfring, J. R. Schultz, F. Nichol, 1973, Biometrics.

Irinotecan therapy in adults with recurrent or progressive malignant glioma.

R. McLendon, A. Friedman, D. Bigner, 1999, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

Corticosteroids in Duchenne muscular dystrophy: Major variations in practice

R. Griggs, S. Pandya, K. Bushby, 2013, Muscle & nerve.

Phase 1 and Pharmacokinetic Study of Intravenous Irinotecan in Refractory Solid Tumor Patients with Hepatic Dysfunction

R. Goldberg, L. Schaaf, M. Rothenberg, 2006, Clinical Cancer Research.

Phase II study of irinotecan plus cisplatin in patients with advanced non-small-cell lung cancer.

David H. Johnson, L. Schaaf, I. Dimery, 1999, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

Phase I and pharmacokinetic trial of oral irinotecan administered daily for 5 days every 3 weeks in patients with solid tumors.

D. V. Von Hoff, M. Villalona-Calero, L. Schaaf, 1999, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

Phase I dose-finding and pharmacokinetic trial of irinotecan hydrochloride (CPT-11) using a once-every-three-week dosing schedule for patients with advanced solid tumor malignancy.

J. Sloan, J. Sloan, R. Goldberg, 2000, Clinical cancer research : an official journal of the American Association for Cancer Research.

Ataluren treatment of patients with nonsense mutation dystrophinopathy

G. Comi, R. Finkel, B. Wong, 2014, Muscle & nerve.

Irinotecan plus gemcitabine induces both radiographic and CA 19-9 tumor marker responses in patients with previously untreated advanced pancreatic cancer.

J. Hainsworth, Mark R. Green, L. Miller, 2002, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

Safety, Tolerability, and Pharmacokinetics of PTC124, a Nonaminoglycoside Nonsense Mutation Suppressor, Following Single‐ and Multiple‐Dose Administration to Healthy Male and Female Adult Volunteers

S. Peltz, S. Paushkin, L. Miller, 2007, Journal of clinical pharmacology.

PTC124 treatment over 3 months improves pharmacodynamic and clinical parameters in patients with nonsense-mutation-mediated CF

H. Blau, E. Kerem, M. Wilschanski, 2008 .

Ataluren (PTC124) Induces CFTR Protein Expression and Activity in Children with Nonsense Mutation Cystic Fibrosis

F. Vermeulen, I. Sermet-Gaudelus, T. Ajayi, 2010 .

Irinotecan Treatment for Recurrent Malignant Glioma Using an Every-3-Week Regimen

T. Cloughesy, F. Kabbinavar, H. Friedman, 2002, American journal of clinical oncology.

Irinotecan plus fluorouracil/leucovorin for metastatic colorectal cancer: a new survival standard.

L. Saltz, J. Douillard, L. Miller, 2001, The oncologist.

Phase I dose-finding and pharmacokinetic trial of irinotecan (CPT-11) administered every two weeks.

D. V. Von Hoff, M. Villalona-Calero, L. Schaaf, 2001, Annals of oncology : official journal of the European Society for Medical Oncology.

Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial.

S. Peltz, S. McColley, E. Kerem, 2014, The Lancet. Respiratory medicine.

Ataluren (PTC124) induces cystic fibrosis transmembrane conductance regulator protein expression and activity in children with nonsense mutation cystic fibrosis.

F. Vermeulen, I. Sermet-Gaudelus, T. Ajayi, 2010, American journal of respiratory and critical care medicine.

Phase I I S tudy o f I rinotecan P lus C isplatin i n P atients With A dvanced N on-Small-Cell L ung C ancer

L. Schaaf, I. Dimery, J. Garst, 1999 .

Group sequential designs for clinical trials.

G. Elfring, J. R. Schultz, 1973, Biometrics.

A randomized placebo-controlled trial of ataluren for the treatment of nonsense mutation cystic fibrosis

S. Peltz, S. McColley, F. Accurso, 2014 .

Phase 1 Study of Safety, Tolerability, and Pharmacokinetics of PTC299, an Inhibitor of Stress‐Regulated Protein Translation

S. Peltz, Thomas P. Davis, G. Elfring, 2016, Clinical pharmacology in drug development.

T.P.5.01 Phase 2b Study of PTC124 in Duchenne/Becker muscular dystrophy (DMD/BMD): Demographic and other baseline data

R. Finkel, K. Bushby, K. Flanigan, 2008, Neuromuscular Disorders.

Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: A meta‐analysis of disease progression rates in recent multicenter clinical trials

S. Peltz, D. Cox, P. Shieh, 2019, Muscle & nerve.

Assessment of Treatment Effect With Multiple Outcomes in 2 Clinical Trials of Patients With Duchenne Muscular Dystrophy.

Lee-Jen Wei, C. McDonald, G. Elfring, 2020, JAMA network open.

Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy: patient demographics and characteristics from the STRIDE Registry.

E. Mercuri, F. Muntoni, I. Desguerre, 2019, Journal of comparative effectiveness research.

T.P.5 Timed function tests and other physical function outcomes in Ataluren-treated patients with nonsense mutation Duchenne Muscular Dystrophy (nmDMD)

S. Peltz, C. McDonald, G. Elfring, 2014, Neuromuscular Disorders.

Effects of treatment with clindamycin hydrochloride on progression of canine periodontal disease after ultrasonic scaling.

J. Slots, G. Elfring, D. Nielsen, 2000, Veterinary therapeutics : research in applied veterinary medicine.

Irinotecan plus fluorouracil and leucovorin for metastatic colorectal cancer. Irinotecan Study Group.

L. Saltz, C. Blanke, M. Moore, 2000, The New England journal of medicine.

The minor gentamicin complex component, X2, is a potent premature stop codon readthrough molecule with therapeutic potential

S. Peltz, Jairo L. Sierra, H. Ren, 2018, PloS one.

The 6‐minute walk test as a new outcome measure in Duchenne muscular dystrophy

Jay J. Han, Alina Nicorici, R. Abresch, 2010, Muscle & nerve.

Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis

H. Blau, E. Kerem, T. Ajayi, 2011, European Respiratory Journal.

Phase I clinical and pharmacologic study of weekly cisplatin combined with weekly irinotecan in patients with advanced solid tumors.

N. Kemeny, G. Schwartz, L. Saltz, 1998, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

A multicenter, Phase II trial of weekly irinotecan (CPT‐11) in patients with previously treated colorectal carcinoma

J. Hainsworth, C. Geyer, D. V. Von Hoff, 1999, Cancer.

Phase II trial of irinotecan, paclitaxel and carboplatin in patients with previously untreated Stage IIIB/IV nonsmall cell lung carcinoma

M. Socinski, V. Israel, A. Sandler, 2002, Cancer.

Irinotecan (CPT-11) in triplet combinations in patients with advanced non small-cell lung cancer: a review and report of a phase I/II trial.

M. Socinski, V. Israel, A. Sandler, 2001, Clinical lung cancer.

Phase I trial of the combination of irinotecan, paclitaxel, and carboplatin in patients with advanced non-small-cell lung cancer.

M. Socinski, V. Israel, A. Sandler, 2001, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

Effects of colestipol hydrochloride and neomycin sulfate on cholesterol turnover in the rat

W. A. Phillips, G. Elfring, 2006, Lipids.

Evaluating longitudinal therapy effects via the North Star Ambulatory Assessment

Lee-Jen Wei, F. Muntoni, K. Flanigan, 2021, Muscle & nerve.

Deflazacort versus prednisone/prednisolone for maintaining motor function and delaying loss of ambulation: A post HOC analysis from the ACT DMD trial

S. Peltz, P. Shieh, C. McDonald, 2018, Muscle & nerve.

Longitudinal Evaluation of Working Memory in Duchenne Muscular Dystrophy

G. Elfring, P. Trifillis, M. Thangarajh, 2020, Journal of clinical medicine.

The 6‐minute walk test in Duchenne/Becker muscular dystrophy: Longitudinal observations

Jay J. Han, Alina Nicorici, Erik K Henricson, 2010, Muscle & nerve.

THE 6-MINUTE WALK TEST AND OTHER ENDPOINTS IN DUCHENNE MUSCULAR DYSTROPHY: LONGITUDINAL NATURAL HISTORY OBSERVATIONS OVER 48 WEEKS FROM A MULTICENTER STUDY

S. Peltz, R. Abresch, C. McDonald, 2013, Muscle & nerve.

Phase 2a Study of Ataluren-Mediated Dystrophin Production in Patients with Nonsense Mutation Duchenne Muscular Dystrophy

R. Finkel, B. Wong, S. Peltz, 2013, PloS one.

Percent-Predicted 6-Minute Walk Distance in Duchenne Muscular Dystrophy to Account for Maturational Influences

Jay J. Han, Alina Nicorici, Gary Elfring, 2012, PLoS currents.

PP3.3 – 1808 Disease-related symptoms and activities of daily living: a novel survey of patients with nonsense mutation Duchenne muscular dystrophy

G. Elfring, A. Reha, R. Spiegel, 2013 .

Phase II trial of irinotecan in patients with progressive or rapidly recurrent colorectal cancer.

D. V. Von Hoff, S. Hilsenbeck, H. Burris, 1996, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial

Eitan Kerem, B. Kerem, H. Blau, 2008, The Lancet.

THE 6-MINUTE WALK TEST AND OTHER CLINICAL ENDPOINTS IN DUCHENNE MUSCULAR DYSTROPHY: RELIABILITY, CONCURRENT VALIDITY, AND MINIMAL CLINICALLY IMPORTANT DIFFERENCES FROM A MULTICENTER STUDY

S. Peltz, R. Abresch, C. McDonald, 2013, Muscle & nerve.

Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy.

Lee-Jen Wei, G. Comi, R. Finkel, 2020, Journal of comparative effectiveness research.

Two studies evaluating irinotecan treatment for recurrent malignant glioma using an every‐3‐week regimen

T. Cloughesy, F. Kabbinavar, H. Friedman, 2003, Cancer.

Curve fitting and modeling in pharmacokinetics: A response

G. Elfring, C. Metzler, 1978, Journal of Pharmacokinetics and Biopharmaceutics.

Results of North Star Ambulatory Assessments in the Phase 3 Ataluren Confirmatory Trial in Patients with Nonsense Mutation Duchenne Muscular Dystrophy (ACT DMD)

S. Peltz, F. Muntoni, K. Bushby, 2016 .

Ambulatory quantitative waking and sleeping cough assessment in patients with cystic fibrosis.

Temitayo Ajayi, Eitan Kerem, S. Peltz, 2011, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

Quantitative cough assessment in cystic fibrosis (CF)

S. Peltz, E. Kerem, M. Wilschanski, 2008 .

Following Single-and Multiple-Dose Administration to Healthy Male and Female Adult Safety , Tolerability , and Pharmacokinetics of PTC 124 , a Nonaminoglycoside Nonsense Mutation

S. Peltz, S. Paushkin, L. Miller, 2007 .

P4.10 The relationship of ataluren plasma concentration and response across clinical studies in nonsense mutation dystrophinopathy

R. Finkel, B. Wong, S. Peltz, 2011, Neuromuscular Disorders.

P3.51 Results of a Phase 2b, dose-ranging study of ataluren (PTC124®) in nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD)

R. Finkel, B. Wong, S. Peltz, 2010, Neuromuscular Disorders.

G.P.3.05 Phase 2 study of PTC124 for nonsense mutation suppression therapy of Duchenne muscular dystrophy (DMD)

R. Finkel, B. Wong, K. Flanigan, 2007, Neuromuscular Disorders.

M.P.3.05 Use of a novel system for grading timed function test performance in phase 2b study of ataluren (PTC124™) in nonsense mutation Duchenne and Becker muscular dystrophy (nmDMD/BMD)

R. Abresch, G. Elfring, A. Reha, 2009, Neuromuscular Disorders.

M.P.3.04 Assessment of StepWatch™ activity monitoring in phase 2b study of ataluren (PTC124™) in nmDMD/BMD

R. Abresch, C. McDonald, G. Elfring, 2009, Neuromuscular Disorders.

Biopharmaceutical applications of group sequental designs

G. Elfring, J. R. Schultz, A. E. Hearron, 1984 .

41* PTC124 activity in CF patients carrying stop mutations: results of a phase 2 study

H. Blau, E. Kerem, M. Wilschanski, 2007 .

Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy.

S. Peltz, S. Apkon, C. Campbell, 2021, Journal of comparative effectiveness research.

Phase I study of adozelesin (U-73,975) in patients with solid tumors.

D. Alberts, C. Wiggins, G. Elfring, 1994, Anti-cancer drugs.

The relationship between deficit in digit span and genotype in nonsense mutation Duchenne muscular dystrophy

S. Peltz, G. Elfring, P. Trifillis, 2018, Neurology.

Phase I dose-finding and pharmacokinetic trial of irinotecan hydrochloride (CPT-11) using a once-every-three-week dosing schedule for patients with advanced solid tumor malignancy.

J. Sloan, R. Goldberg, H. Pitot, 2000, Clinical cancer research : an official journal of the American Association for Cancer Research.