Peer-Reviewed Manuscripts

This section contains information and links to select peer-reviewed manuscripts sponsored by Ascendis Pharma. Materials may include information regarding investigational products or uses that have not received FDA approval and therefore safety and efficacy have not been established. Ascendis Pharma does not recommend or endorse using its products in any manner that is inconsistent with FDA-approved labeling.

Endocrine Rare Diseases

Disease State

Products

Growth Hormone Deficiency

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.

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.

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.

Hypoparathyroidism

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.

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.

Achondroplasia

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.

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

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.

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.