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Hormone Research in Paediatrics

Publication date: March 6, 2025 doi. 1159/000545064.

Continued Improvements in Lower Extremity Alignment in Navepegritide-Treated Children With Achondroplasia: Week 104 Results From the ApproaCH Trial

Effects of Navepegritide and Lonapegsomatropin on Manifestations of Skeletal Dysplasia in Children With Achondroplasia: 52-Week Results From the Phase 2 COACH Trial

Population Pharmacokinetics and Exposure Response Analyses of Navepegritide in Children With Achondroplasia

Navepegritide Combined With Lonapegsomatropin for the Treatment of Children With Achondroplasia: 52-Week Results From the Phase 2 COACH Trial

ApproaCH Trial Open Label Extension: Week 104 Results in Children With Achondroplasia Aged 5 Years or Older Treated With Navepegritide

Navepegritide Combined With Lonapegsomatropin for the Treatment of Children With Achondroplasia: 52-Week Results from the Phase 2 COACH Trial

McDonnell CM, Irving M, Nolting LA, et al. Eur J Endocrinol. 2026;194(6):745-755. doi:10.1093/ejendo/lvag082

Health-Related Quality of Life in Hypoparathyroidism

Palopegteriparatide Enhances Work Functioning and Productivity in Employed Adults With Chronic Hypoparathyroidism: 156-Week Results From the PaTHway Trial

Palopegteriparatide Treatment in Adults With Hypoparathyroidism: Final Results of the Phase 3 PaTHway Trial

Five-Year Data Report From the Phase 2 PaTH Forward Trial of Palopegteriparatide for Adults With Hypoparathyroidism

Safety and Effectiveness of Lonapegsomatropin in the SkybriGHt Registry: Analysis of the First 100 Patients Enrolled With 2 Years of Follow-Up

Idiopathic Short Stature Overview

Small for Gestational Age Without Catch-Up Growth Overview

Turner Syndrome Overview

SHOX Deficiency Overview

Long-Term Safety and Efficacy of Lonapegsomatropin in Adults With Growth Hormone Deficiency: Results From a 52-Week Open-Label Extension Trial

Hypoparathyroidism and Renal Function

Growth Outcomes and Safety of Navepegritide in Children With Achondroplasia: Results of the ApproaCH Trial Open-Label Extension

Sustained Improvement in Renal Function With Palopegteriparatide in Adults With Chronic Hypoparathyroidism: Three-Year Results From the PaTH Forward and PaTHway Trials

Longitudinal Observation of Children With Achondroplasia: Findings From a Global Natural History Study (ACHieve)

McDonnell C, Hove HB, Irving M, et al. Horm Res Paediatr. Published online January 8, 2026. doi:10.1159/000550169.

Palopegteriparatide for Adults With Chronic Hypoparathyroidism: Skeletal Dynamics Through 3 Years of the Phase 2 PaTH Forward Trial

Rubin MR, Clarke BL, Hofbauer LC, et al. J Bone Miner Res. 2026;zjag013. doi: 10.1093/jbmr/zjag013.

Patient-Reported Outcomes in Palopegteriparatide-Treated Adults With Hypoparathyroidism: PaTH Forward Trial Extension

Rubin M, Palermo A, Vokes T, et al. J Clin Endocrinol Metab. 2025;dgaf653. doi: 10.1210/clinem/dgaf653.

Efficacy and Safety of Once-Weekly Lonapegsomatropin in Adults With Growth Hormone Deficiency: foresiGHt Trial Results

Biller BMK, Gilis-Januszewska A, Doknic M, et al. J Clin Endocrinol Metab. 2025;dgaf680. doi: 10.1210/clinem/dgaf680.

Prevalence, Treatment Patterns, and Characteristics of US Adults With Confirmed or at Risk for Growth Hormone Deficiency

Hoffman AR, Raveendran S, Manjelievskaia J, et al. Adv Ther. 2025;42(6):2853-2873. doi: 10.1007/s12325-025-03188-6.

Once-Weekly Navepegritide in Children With Achondroplasia: The ApproaCH Randomized Clinical Trial

Savarirayan R, McDonnell C, Bacino CA, et al. JAMA Pediatr. 2026;180(1):18-25. doi:10.1001/jamapediatrics.2025.4771.

Achondroplasia Overview

Children With Growth Hormone Deficiency Treated With Lonapegsomatropin Demonstrated Sustained Height Improvements for up to 6 Years: enliGHten Trial Final Results

Maniatis AK. Thornton PS, Nadgir UM, et al. Horm Res Paediatr. 2025;6:1-13. doi: 10.1159/000545064.

Switching to Weekly Lonapegsomatropin from Daily Somatropin in Children With Growth Hormone Deficiency: The fliGHt Trial

Maniatis AK, Nadgir U, Saenger P, et al. Horm Res Paediatr. 2022;95(3):233-243. doi: 10.1159/000524003.

Safety and Efficacy of Lonapegsomatropin in Children With Growth Hormone Deficiency: enliGHten Trial 2-Year Results

Maniatis AK, Casella SJ, Nadgir UM, et al. J Clin Endocrinol Metab. 2022;107(7):e2680-e2689. doi: 10.1210/clinem/dgac217.

Average IGF-1 Prediction for Once-Weekly Lonapegsomatropin in Children With Growth Hormone Deficiency

Lin Z, Shu AD, Bach M, et al. J Endocr Soc. 2021;6(1):bvab168. doi:10.1210/jendso/bvab168.

Weekly Lonapegsomatropin in Treatment-Naïve Children With Growth Hormone Deficiency: The Phase 3 heiGHt Trial

Thornton PS, Maniatis AK, Aghajanova E, et al. J Clin Endocrinol Metab. 2021;106(11):3184-3195. doi: 10.1210/clinem/dgab529.

The Rationale and Design of TransCon Growth Hormone for the Treatment of Growth Hormone Deficiency

Sprogøe K, Mortensen E, Karpf DB, et al. Endocr Connect. 2017;6(8):R171-R181. doi: 10.1530/EC-17-0203.

A Phase 2 Trial of Long-Acting TransCon Growth Hormone in Adult GH Deficiency

Höybye C, Pfeiffer AFH, Ferone D, et al. Endocr Connect. 2017;6(3):129-138. doi: 10.1530/EC-17-0007.

A Randomized Phase 2 Study of Long-Acting TransCon GH vs Daily GH in Childhood GH Deficiency

Chatelain P, Malievskiy O, Radziuk K, et al. J Clin Endocrinol Metab. 2017;102(5):1673-1682. doi:10.1210/jc.2016-3776.

Once-Weekly TransCon CNP (Navepegritide) in Children With Achondroplasia (ACcomplisH): A Phase 2, Multicentre, Randomised, Double-Blind, Placebo-Controlled, Dose-Escalation Trial

Savarirayan R, Hoernschemeyer DG, Ljungberg M, et al. eClinicalMedicine. 2023;65:102258. doi:10.1016/j.eclinm.2023.102258.

Phase 1 Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Results of a Long-Acting C-Type Natriuretic Peptide Prodrug, TransCon CNP

Breinholt VM, Mygind PH, Christoffersen ED, et al. Br J Clin Pharmacol. 2022;88(11):4763-4772. doi:10.1111/bcp.15369.

Early U.S. Real-World Treatment Patterns and Outcomes in Palopegteriparatide Treatment for Patients With Hypoparathyroidism

Graham T, Shoback DM, Abbott L, et al. Endocr Pract. 2025;S1530-891X(25)01003-1. doi:10.1016/j.eprac.2025.08.008.

Palopegteriparatide Treatment Improves Renal Function in Adults With Chronic Hypoparathyroidism: 1-Year Results From the Phase 3 PaTHway Trial

Rejnmark L, Gosmanova EO, Khan AA, et al. Adv Ther. 2024;41(6):2500-2518. doi:10.1007/s12325-024-02843-8.

Efficacy and Safety of TransCon PTH in Adults With Hypoparathyroidism: 52-Week Results From the Phase 3 PaTHway Trial

Clarke BL, Khan AA, Rubin MR, et al. J Clin Endocrinol Metab. 2024;110(4):951-960. doi:10.1210/clinem/dgae693.

Efficacy and Safety of Parathyroid Hormone Replacement With TransCon PTH in Hypoparathyroidism: 26-Week Results From the Phase 3 PaTHway Trial

Khan AA, Rubin M, Schwarz P, et al. J Bone Miner Res. 2023;38(1):14-25. doi: 10.1002/jbmr.4726.

PaTH Forward: A Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial of TransCon PTH in Adult Hypoparathyroidism

Khan AA, Rejnmark L, Rubin M, et al. J Clin Endocrinol Metab. 2022;107(1):e372-e385. doi: 10.1210/clinem/dgab577.

A Randomized Double-Blind Placebo-Controlled First-In-Human Phase 1 Trial of TransCon PTH in Healthy Adults

Karpf DB, Pihl S, Mourya S, et al. J Bone Miner Res. 2020;35(8):1430-1440. doi: 10.1002/jbmr.4016.

Design and Preclinical Development of TransCon PTH, an Investigational Sustained‐Release PTH Replacement Therapy for Hypoparathyroidism

Holten-Andersen L, Pihl S, Rasmussen CE, et al. J Bone Miner Res. 2019;34(11):2075-2086. doi: 10.1002/jbmr.3824.

Adult Growth Hormone Deficiency Overview

Pediatric Growth Hormone Deficiency Overview

Hypoparathyroidism Overview

Once-Weekly Lonapegsomatropin (TransCon hGH) Added to Once-Weekly Navepegritide (TransCon CNP) in Children With Achondroplasia: 26-Week Results from the Phase 2 COACH Trial

Safety and Effectiveness of Lonapegsomatropin in the SkybriGHt Registry Study: Analysis of the First 100 Patients Enrolled With 1 Year of Follow-Up

Results of the foresiGHt Trial Support the Efficacy and Safety of Once-Weekly Lonapegsomatropin in Adults With Growth Hormone Deficiency

The Majority of Children With Pediatric Growth Hormone Deficiency Treated With Lonapegsomatropin for up to 6 Years Met or Exceeded Average Parental Height SDS: Final Results of enliGHten

Practical Management of Pediatric Growth Hormone Deficiency With Lonapegsomatropin-tcgd

Design of the New InsiGHTS Trial: A Multicenter, Phase 2, Randomized, Open-Label, Active Controlled Study to Investigate the Safety, Tolerability, and Efficacy of Lonapegsomatropin-tcgd in Prepubertal Individuals With Turner Syndrome

Improvements in Lower Extremity Alignment are Associated With Physical Functioning in Children With Achondroplasia Treated With Navepegritide: 52-Week Results From the ApproaCH Trial

TransCon CNP (Navepegritide) Improved Physical Functioning in Children With Achondroplasia in the ApproaCH Trial

Improved Skeletal Dynamics in Adults Treated With Palopegteriparatide for Chronic Hypoparathyroidism: 214-Week Results From the Phase 2 PaTH Forward Trial

Efficacy and Safety of Palopegteriparatide Treatment in Adults With Hypoparathyroidism: 3-Year Results From the Phase 3 PaTHway Trial

Sustained Improvement in Skeletal Dynamics and Renal Function With Palopegteriparatide in Adults With Chronic Hypoparathyroidism: 2-Year Results From the Phase 3 PaTHway Trial

Long-Term Efficacy and Safety of Palopegteriparatide Treatment in Adults With Chronic Hypoparathyroidism: 4-Year Results From the Phase 2 PaTH Forward Trial

Sustained Improvement in Renal Function vith Palopegteriparatide in Adults With Chronic Hypoparathyroidism: 2-Year Results From the Phase 3 PaTHway Trial

TransCon PTH Enables Independence From Conventional Therapy While Maintaining Normal Serum Calcium in Adults With Chronic Postsurgical Hypoparathyroidism: Results From a Sub-Analysis of the PaTHway Phase 3 Trial