Results 11 to 20 of about 5,458 (150)

Terlipressin for Hepatorenal Syndrome in Patients With Early-Stage Acute-on-Chronic Liver Failure. [PDF]

Liver Int
ABSTRACT Background & Aims Hepatorenal syndrome‐acute kidney injury (HRS‐AKI) is a life‐threatening complication of decompensated cirrhosis. The US Food and Drug Administration approved terlipressin use for HRS‐AKI based on the CONFIRM study, which demonstrated a significant improvement in HRS reversal with terlipressin versus placebo.
Rockey DC, Gordon F, Thuluvath PJ, Victor D, Kemmer N, Cardoza S, Jamil K, Frederick RT.   +7 more
europepmc   +2 more sources

Maralixibat Reduces Serum Bile Acids and Improves Cholestatic Pruritus in Adolescents With Alagille Syndrome. [PDF]

Liver Int
ABSTRACT Background Alagille syndrome (ALGS) is a multisystem cholestatic disorder. Maralixibat is approved for the treatment of cholestatic pruritus in ALGS with limited data in adults. Methods Participants were included if they received ≥ 2 doses of maralixibat at age ≥ 16 years in one of the three previously published maralixibat ALGS clinical ...
Hirschfield G, Vandriel SM, Mogul DB, Baek M, Vig P, Kamath BM.   +5 more
europepmc   +2 more sources

Phase II clinical trial of nirogacestat in patients with relapsed ovarian granulosa cell tumours. [PDF]

Clin Transl Med
Evidence suggests NOTCH activation is among the survival and proliferation pathways interacting with FOXL2 c.402C > G (p.Cys134Trp) mutation in granulosa cell tumours (GCT). This Phase II clinical trial of nirogacestat in GCT achieved its enrolment target in < 1 year and primary analysis within 2 years.
Grisham RN, Hopp E, Pennington K, Holloway R, Wenham RM, Blecharz P, Dockery L, Matsuo K, Salani R, Bidzinski M, Braly P, Celano P, Reid T, Seward S, Lewis J, Johnson M, DuBose R, Ahn S, Cheng S, Alvero C, Konstantinopoulos PA.   +20 more
europepmc   +2 more sources

Bradycardia and Other Arrhythmias in Patients With Hepatorenal Syndrome-Acute Kidney Injury Following Terlipressin Treatment: A Pooled Analysis of Three North American Phase III Clinical Studies. [PDF]

Aliment Pharmacol Ther
In a pooled analysis, bradycardia was observed in 6% of terlipressin‐treated patients but rarely required a dose interruption or reduction. Arrhythmia serious adverse event incidences were low and similar in both treatment groups. Routine intensive cardiac monitoring for the detection of arrhythmias may not be necessary during terlipressin ...
Bajaj JS, Kwo P, Pappas SC, O'Leary JG, Jamil K, Cardoza S, Wong F.   +6 more
europepmc   +2 more sources

A model of academic-practice collaboration for facilitating informatics capacity and building a learning health system framework in public health. [PDF]

Learn Health Syst
Abstract Background and Objective The data modernization initiative (DMI) is a multi‐year, multi‐billion‐dollar endeavor toward a robust public health information infrastructure. The various DMI projects (interoperability, analytics, workforce, governance) present an opportunity for a learning health system (LHS) framework in public health.
Rajamani S, Solarz S, Muscoplat MH, Schmit AD, Gonderinger A, Brueske C, Fritz J, Emerson E, Melton GB.   +8 more
europepmc   +2 more sources

Psychometric evaluation of the PROMIS parent proxy mobility item bank for use in Duchenne muscular dystrophy. [PDF]

Dev Med Child Neurol
Abstract Aim To evaluate the psychometric properties and measurement quality of the Patient‐Reported Outcomes Measurement Information System Parent Proxy (PROMIS PP) Mobility item bank (v1.0, 23 items) for children with Duchenne muscular dystrophy (DMD), through Rasch statistical analysis. Method De‐identified PROMIS PP Mobility items were completed by
Lowes LP, Le Reun CM, Alfano LN, Reash NF, Iammarino MA, Patel S, Audhya IF.   +6 more
europepmc   +2 more sources

B cell and aquaporin-4 antibody relationships with neuromyelitis optica spectrum disorder activity. [PDF]

Ann Clin Transl Neurol
Abstract This post hoc analysis of the randomized, placebo‐controlled N‐MOmentum study (NCT02200770) of inebilizumab in neuromyelitis optica spectrum disorder (NMOSD) evaluated relationships between circulating B‐cell subsets and aquaporin‐4 immunoglobulin G (AQP4‐lgG) titers and attacks.
Bennett JL, Pittock SJ, Paul F, Kim HJ, Irani SR, O'Connor KC, Patterson KR, Smith MA, Gunsior M, Mittereder N, Rees WA, Cimbora D, Cree BAC.   +12 more
europepmc   +2 more sources

Defining Renal Recovery in Patients With Hepatorenal Syndrome-Acute Kidney Injury: Experience From North American Studies. [PDF]

JGH Open
ABSTRACT Introduction The degree of improvement in serum creatinine (SCr) has previously been suggested as a sensitive indicator of treatment response in patients with hepatorenal syndrome‐acute kidney injury (HRS‐AKI), while HRS reversal remains the primary endpoint in clinical trials.
Mujtaba MA, Elsiesy H, Faiz S, Hussain SA, Gamilla-Crudo AKN, Karim A, Khan MI, Khattak MW, Zafar Z, Kueht M, Jamil K.   +10 more
europepmc   +2 more sources

Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to <8 Years) with Duchenne Muscular Dystrophy: 1‐Year Interim Results from Study SRP‐9001‐103 (ENDEAVOR)

Annals of Neurology, Volume 94, Issue 5, Page 955-968, November 2023., 2023
Objective Delandistrogene moxeparvovec is approved in the USA for the treatment of ambulatory patients (4–5 years) with Duchenne muscular dystrophy. ENDEAVOR (SRP‐9001‐103; NCT04626674) is a single‐arm, open‐label study to evaluate delandistrogene moxeparvovec micro‐dystrophin expression, safety, and functional outcomes following administration of ...
Craig M. Zaidman, Crystal M. Proud, Craig M. McDonald, Kelly J. Lehman, Natalie L. Goedeker, Stefanie Mason, Alexander P. Murphy, Maitea Guridi, Shufang Wang, Carol Reid, Eddie Darton, Christoph Wandel, Sarah Lewis, Jyoti Malhotra, Danielle A. Griffin, Rachael A. Potter, Louise R. Rodino‐Klapac, Jerry R. Mendell   +17 more
wiley   +1 more source

Preclinical development and characterization of a human plasma‐derived high‐purity factor X concentrate for therapeutic use

Haemophilia, Volume 29, Issue 6, Page 1394-1409, November 2023., 2023
Abstract Introduction Hereditary factor X deficiency is a rare bleeding disorder, with limited treatment options. This paper describes the approach to pre‐clinical development and characterization of a high‐purity plasma‐derived factor X concentrate, to achieve orphan drug marketing authorization for the treatment of hereditary factor X deficiency ...
Joanne Lloyd, Peter Feldman
wiley   +1 more source