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Quality of life improvements following one year of setmelanotide in children and adult patients with Bardet-Biedl syndrome: phase 3 trial results

Orphanet journal of rare diseases, 2023-01, Vol.18 (1), p.12-12, Article 12

2023

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Sleep and physical activity patterns in adults and children with Bardet–Biedl syndrome

Orphanet journal of rare diseases, 2021-06, Vol.16 (1), p.1-276, Article 276

2021

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The efficacy and safety of setmelanotide in individuals with Bardet-Biedl syndrome or Alström syndrome: Phase 3 trial design

Contemporary clinical trials communications, 2021-06, Vol.22, p.100780-100780, Article 100780

2021

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Efficacy and safety of setmelanotide, a melanocortin-4 receptor agonist, in patients with Bardet-Biedl syndrome and Alström syndrome: a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial with an open-label period

The lancet. Diabetes & endocrinology, 2022-12, Vol.10 (12), p.859-868

2022

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Early development and adaptive functioning in children with Bardet‐Biedl syndrome

American journal of medical genetics. Part A, 2024-01, Vol.194 (1), p.31-38

2024

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Kidney failure in Bardet–Biedl syndrome

Clinical genetics, 2022-04, Vol.101 (4), p.429-441

2022

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Cutaneous findings in Bardet‐Biedl syndrome

International journal of dermatology, 2019-10, Vol.58 (10), p.1160-1164

2019

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Interview-Based Patient- and Caregiver-Reported Experiences of Hunger and Improved Quality of Life with Setmelanotide Treatment in Bardet-Biedl Syndrome

Advances in therapy, 2023-05, Vol.40 (5), p.2394-2411

2023

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An international cohort study of autosomal dominant tubulointerstitial kidney disease due to REN mutations identifies distinct clinical subtypes

Kidney international, 2020-12, Vol.98 (6), p.1589-1604

2020

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Bardet‐Biedl syndrome: Weight patterns and genetics in a rare obesity syndrome

Pediatric obesity, 2021-02, Vol.16 (2), p.e12703-n/a

2021

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Thoraco-Abdominal Abnormalities in Bardet-Biedl Syndrome: Situs Inversus and Heterotaxy

The Journal of pediatrics, 2019-01, Vol.204, p.31-37

2019

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Renal transplantation in Bardet–Biedl Syndrome

Pediatric nephrology (Berlin, West), 2016-11, Vol.31 (11), p.2153-2161

2016

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Diarrhea in a post-renal transplant child: Questions

Pediatric nephrology (Berlin, West), 2015-04, Vol.30 (4), p.589-590

2015

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Diarrhea in a post-renal transplant child: Answers

Pediatric nephrology (Berlin, West), 2015-04, Vol.30 (4), p.591-593

2015

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Bardet–Biedl syndrome: A model for translational research in rare diseases

European journal of molecular and clinical medicine, 2015-05, Vol.2 (4-5), p.102-109

2015

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Reply

The Journal of pediatrics, 2020-03, Vol.218, p.264

2020

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Efficacy of albumin and diuretic therapy in children with nephrotic syndrome

Pediatrics (Evanston), 1993-06, Vol.91 (6), p.1142-1146

1993

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An international cohort study of autosomal dominant tubulointerstitial kidney disease due to mutations identifies distinct clinical subtypes

Kidney international, 2020-12, Vol.98 (6), p.1589-1604

2020

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Reply

The Journal of pediatrics, 2020-03, Vol.218, p.264

2020

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Reply

The Journal of pediatrics, 2020-03, Vol.218, p.264-264

2020

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Childhood pyelonephritis: comparison of gadolinium-enhanced MR imaging and renal cortical scintigraphy for diagnosis

Radiology, 1998-05, Vol.207 (2), p.377-384

1998

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Glomerular Losartan (DuP 753)-sensitive angiotensin II receptor density is increased in young spontaneously hypertensive rats

Pediatric research, 1994-06, Vol.35 (6), p.671-676

1994

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Spontaneous remission of congenital nephrotic syndrome a case report and review of the literature

Pediatric nephrology (Berlin, West), 1992, Vol.6 (1), p.82-84

1992

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Facilitated transport from ternary cation mixtures through water—chloroform—water membrane systems containing macrocyclic ligands

Journal of membrane science, 1984-01, Vol.20 (3), p.273-284

1984

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Effects of the Novel Long-Acting GLP-1 Agonist, Albiglutide, on Cardiac Function, Cardiac Metabolism, and Exercise Capacity in Patients With Chronic Heart Failure and Reduced Ejection Fraction

JACC. Heart failure, 2016-07, Vol.4 (7), p.559-566

2016

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Quality of life improvements following one year of setmelanotide in children and adult patients with Bardet-Biedl syndrome: phase 3 trial results

Sleep and physical activity patterns in adults and children with Bardet–Biedl syndrome

The efficacy and safety of setmelanotide in individuals with Bardet-Biedl syndrome or Alström syndrome: Phase 3 trial design

Efficacy and safety of setmelanotide, a melanocortin-4 receptor agonist, in patients with Bardet-Biedl syndrome and Alström syndrome: a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial with an open-label period

Early development and adaptive functioning in children with Bardet‐Biedl syndrome

Kidney failure in Bardet–Biedl syndrome

Cutaneous findings in Bardet‐Biedl syndrome

Interview-Based Patient- and Caregiver-Reported Experiences of Hunger and Improved Quality of Life with Setmelanotide Treatment in Bardet-Biedl Syndrome

An international cohort study of autosomal dominant tubulointerstitial kidney disease due to REN mutations identifies distinct clinical subtypes

Bardet‐Biedl syndrome: Weight patterns and genetics in a rare obesity syndrome

Thoraco-Abdominal Abnormalities in Bardet-Biedl Syndrome: Situs Inversus and Heterotaxy

Renal transplantation in Bardet–Biedl Syndrome

Diarrhea in a post-renal transplant child: Questions

Diarrhea in a post-renal transplant child: Answers

Bardet–Biedl syndrome: A model for translational research in rare diseases

Reply

Efficacy of albumin and diuretic therapy in children with nephrotic syndrome

An international cohort study of autosomal dominant tubulointerstitial kidney disease due to mutations identifies distinct clinical subtypes

Reply

Reply

Childhood pyelonephritis: comparison of gadolinium-enhanced MR imaging and renal cortical scintigraphy for diagnosis

Glomerular Losartan (DuP 753)-sensitive angiotensin II receptor density is increased in young spontaneously hypertensive rats

Spontaneous remission of congenital nephrotic syndrome a case report and review of the literature

Facilitated transport from ternary cation mixtures through water—chloroform—water membrane systems containing macrocyclic ligands

Effects of the Novel Long-Acting GLP-1 Agonist, Albiglutide, on Cardiac Function, Cardiac Metabolism, and Exercise Capacity in Patients With Chronic Heart Failure and Reduced Ejection Fraction

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