Publications - Ascendis Pharma US

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TransCon^® hGH (lonapegsomatropin)

Lonapegsomatropin (TransCon hGH) in Children with Growth Hormone Deficiency: Efficacy and Safety of up to 2 Years of Treatment
Presented at the PES 2021 Virtual Annual Meeting, April 2021.
A. K. Maniatis, U. M. Nadgir, P. Hofman, P. Saenger, E. D. Chertok, E. M. Aghajanova, W. Song, M. Mao, S. Chessler, A. Komirenko, M. Beckert, A. D. Shu, P. S. Thornton

Estimating the Weekly Average IGF-1 from a Single IGF-1 Sample in Children with GHD Treated with Lonapegsomatropin
Presented at the PES 2021 Virtual Annual Meeting, April 2021.
Z. Lin, Y. Su, S. Chessler, A. Komirenko, E. Dam Christoffersen, A. D. Shu

Continued Efficacy and Safety with up to 2 Years of Treatment with Lonapegsomatropin (TransCon™️ hGH) in Children with Growth Hormone Deficiency
Presented at the PENS 2021 Virtual National Conference, April 2021.
D. Campbell, K. Walsh, N. Marlen, W. Song, M. Mao, S. Chessler, A. Komirenko, M. Beckert, A. D. Shu

Efficacy and Safety of up to 2 Years of Treatment with TransCon hGH (lonapegsomatropin) in Treatment-Naïve and Treatment-Experienced Children with Growth Hormone Deficiency
Presented at the ENDO 2021, March 2021.
A.K. Maniatis, S.J. Casella, U.M. Nadgir, P. Hofman, P. Saenger, E.D. Chertok, E.M. Aghajanova, W. Song, M. Mao, S. Chessler, A. Komirenko, M. Beckert, A.D. Shu, P.S. Thornton.

Design of the foresiGHt Trial: A Multicenter, Randomized, Placebo- and Active-Controlled Trial to Compare Once-Weekly TransCon hGH (lonapegsomatropin) to Placebo and Daily Somatropin in Adults with Growth Hormone Deficiency (GHD)
Presented at the ENDO 2021, March 2021.
M. Fleseriu, J.O.L. Jørgensen, K.C.J. Yuen, C. Höybye, M. Mao, J. Kang, W. Song, A. Komirenko, A.D. Shu, M. Beckert.

Economic Burden of Growth Hormone Deficiency in a US Pediatric Population
Presented at the ENDO 2021, March 2021.
P. Kaplowitz, J. Manjelievskaia, L. Lopez-Gonzalez, C.D. Morrow, P. Pitukcheewanont, A. Smith.

Treatment Experience of Children with Growth Hormone Deficiency in the Phase 3 fliGHt Trial: Switching from Daily Growth Hormone to Once-weekly TransCon hGH
Presented at the Pediatric Endocrinology Nursing Society (PENS) 2020 national conference (virtual)
November 2 – 5, 2020
D. Campbell, K. Walsh, C. Herz, L. Weber, N. Marlen, K. Kummer, L. Reys, J. Gianettoni, J. Peng, A.D. Shu

Introduction of a Novel GH Auto-Injector for Once-weekly Administration of TransCon hGH
Presented at the Pediatric Endocrinology Nursing Society (PENS) 2020 national conference (virtual)
November 2 – 5, 2020
K. Walsh, D. Campbell, J. Permuy, M. Henson, L. Rodriguez, B. Macchus, A. Shiver, S. Jensen, A. Shu

Phase 3 fliGHt Trial: Experience of Switching from Daily Growth Hormone Therapy to Once-weekly TransCon hGH in Children with Growth Hormone Deficiency
Presented at the ENDO Online 2020 virtual meeting
June 8 – 22, 2020
A.K. Maniatis, U. Nadgir, P. Saenger, G. Mick, K.L. Reifschneider, M.J. Abuzzahab, L. Deeb, L.A. Fox, K.A. Woods, J.M. Peng, D.B. Karpf, A.D. Shu, W. Song, S.J. Casella, P.S. Thornton

Maintenance of Favorable Treatment Effect of Once-Weekly TransCon hGH for Children with Growth Hormone Deficiency: Interim Analysis from the enliGHten Long-Term Extension Trial
Abstract presented at the ENDO Online 2020 virtual meeting
June 8 – 22, 2020
A.K. Maniatis, S.J. Casella, U.M. Nadgir, G.J. Mick, P. Hofman, P. Saenger, E.D. Chertok, Warshauer, W. Song, J.M. Peng, A.S. Komirenko, A.D. Shu, M. Beckert, E.M. Aghajanova, P.S. Thornton

The Pivotal Phase 3 heiGHt Trial of Weekly TransCon hGH vs. Daily hGH in Treatment-Naive Subjects with Pediatric Growth Hormone Deficiency
Poster presented at the Pediatric Academic Societies/Pediatric Endocrine Society Annual Meeting (PAS/PES)
April 27 – 30, 2019 (Baltimore, MD)
A.K. Maniatis, P. Thornton, P. Hofman, E. Aghajanova, E. Chertok, M. Korpal-Szczyrska, E. Giorgadze, T. Kovalenko, A.D. Shu, D.B. Karpf, W. Song, M. Beckert, J.A. Leff

TransCon hGH as a Long-Acting Growth Hormone for the Treatment of Pediatric Growth Hormone Deficiency
Poster presented at the Pediatric Endocrinology Nursing Society (PENS) 2019 National Conference
April 25 – 28, 2019 (Long Beach, CA)
D.L. Campbell, K.J. Walsh, C. Herz, J.W. Permuy, D.B. Karpf, W. Song, M. Beckert, J.A. Leff, A.D. Shu

TransCon^® Growth Hormone in the Treatment of Pediatric Growth Hormone Deficiency: Results of the Phase 3 heiGHt Trial
Presented at the Endocrine Society (ENDO) Annual Meeting and Expo
March 23 – 26, 2019 (New Orleans, LA)
P. Thornton, P. Hofman, A.K. Maniatis, E. Aghajanova, E. Chertok, M. Korpal-Szczyrska, E. Giorgadze, T. Kovalenko, A.D. Shu, D.B. Karpf, M. Beckert, J.A. Leff

Baseline Demographics of the TransCon Growth Hormone Phase 3 heiGHt Trial
Poster presented at the 57th Annual Meeting of the European Society for Paediatric Endocrinology (ESPE)
September 27 – 29, 2018 (Athens, Greece)
M. Beckert, A. Shu, D.B. Karpf, A. Maniatis, Z. Lin, J. A. Leff

The Rationale and Design of TransCon GH
Poster presented at the 57th Annual Meeting of the European Society for Paediatric Endocrinology (ESPE)
September 27 – 29, 2018 (Athens, Greece)
K. Sprogøe, A. Shu, M. Beckert, E. Mortensen, D.B. Karpf, J.A. Leff

Baseline Demographics of the TransCon Growth Hormone Phase 3 heiGHt Trial
Poster presented at the 9th International Congress of the Growth Hormone Research & IGF Societies (GRS)
September 14 – 17, 2018 (Seattle, WA)
A. Shu, M. Beckert, D.B. Karpf, Z. Lin, J. A. Leff

Baseline Demographics of the TransCon Growth Hormone Phase 3 heiGHt Trial
Poster presented at the Endocrine Society (ENDO) Annual Meeting and Expo
March 17 – 20, 2018 (Chicago, IL)
M. Beckert, D.B. Karpf, A. Shu, Z. Lin, J.A. Leff

User Driven Development of a New Device for Weekly Growth Hormone Administration in Pediatric Patients
Poster presented at the 10th International Meeting of Pediatric Endocrinology (IMPE)
September 14 – 17, 2017 (Washington, D.C.)
K.Sprogøe, S. Jensen, H. Egesborg, P.E. Fabricius, B. Sørensen, and S.V. Jacobsen

Development of a Long-Acting Growth Hormone (LAGH): Size Matters
Poster presented at the 10th International Meeting of Pediatric Endocrinology (IMPE)
September 14 – 17, 2017 (Washington, D.C.)
P. Saenger, D.B. Karpf, E. Mortensen, M. Beckert, K. Sprogøe, J. Leff

Design and Clinical Development of TransCon Growth Hormone for Growth Hormone Deficiency
Poster presented at the Endocrine Society (ENDO) Annual Meeting and Expo
April 1 – 4, 2017 (Orlando, FL)
M.Beckert, J.M. Mikkelsen, G.N. Rasmussen, H. Rau, K. Sprogøe, J.A. Leff

User Driven Development of a New Device for Weekly Growth Hormone Administration in Pediatric Patients
Poster presented at the Endocrine Society (ENDO) Annual Meeting and Expo
April 1 – 4, 2017 (Orlando, FL)
S. Jensen, H. Egesborg, P.E. Fabricius, B. Sørensen, S.V. Jacobsen

Design of a Bracketed Dosing Regimen for the Administration of TransCon hGH, a Once-Weekly Growth Hormone Product
Poster presented at the Endocrine Society (ENDO) Annual Meeting and Expo
April 1 – 4, 2017 (Orlando, FL)
K. Sprogøe, S. Jensen, H. Egesborg

Design and Rationale for the heiGHt Trial, a Phase 3 TransCon hGH Study in Children with Growth Hormone Deficiency
Poster presented at the Endocrine Society (ENDO) Annual Meeting and Expo
April 1 – 4, 2017 (Orlando, FL)
M. Beckert, D.B. Karpf, E. Mortensen, J. Mardell, E.D. Christoffersen, J.A. Leff

The Rationale and Design of TransCon Growth Hormone for the Treatment of Growth Hormone Deficiency
Endocr Connect. 2017,6(8):R171–R181.
K. Sprogøe, E. Mortensen, D.B. Karpf, J.A. Leff

A Randomized Phase 2 Study of Long-Acting TransCon GH vs. Daily GH in Childhood GH Deficiency
J Clin Endocrinol Metab. 2017, 102(5): 1673–1682; doi: 10.1210/jc.2016-3776.
P. Chatelain, O. Malievsky, K. Radziuk, G. Senatorova, M. Abdou, E. Vlachopapadopolou, Y. Skorodok, V. Peterkova, J. Leff, M. Beckert, the TransCon GH Working Group. J Clin Endocrinol Metab

A Phase 2 Trial of Long-acting TransCon Growth Hormone in Adult GH Deficiency
Endocr Connect. 2017,6(3):129–138.
C. Höybye, A.F.H. Pfeiffer, D. Ferone, J.S. Christiansen, D. Gilfoyle, E.D. Christoffersen, E. Mortensen, J.A. Leff, M. Beckert

A First-in-Man Phase 1 Trial for Long-Acting TransCon Growth Hormone
Epub December 5, 2017
D. Gilfoyle, E. Mortensen, E.D. Christoffersen, J.A. Leff, M. Beckert. Growth Horm IGF Res. 2018

Pediatric Phase 2 Data Demonstrate That TransCon hGH has an Anti-hGH Immunogenic Profile that Is Comparable to Daily hGH
Poster presented at 55th Annual Meeting of the European Society for Paediatric Endocrinology (ESPE)
September 10 – 12, 2016 (Paris, France)
D. Gilfoyle, S. Pihl, P. Chatelain, M. Beckert

A Six-Month Safety and Efficacy Study of TransCon hGH Compared to Daily hGH in Prepubertal Children with Growth Hormone Deficiency (GHD)
Poster presented at 55th Annual Meeting of the European Society for Paediatric Endocrinology (ESPE)
September 10 – 12, 2016 (Paris, France)
P. Chatelain, O. Malievsky, K. Radziuk, G. Senatorova, M. Beckert

Design and Clinical Development of TransCon Growth Hormone for Growth Hormone Deficiency
Poster presented at 55th Annual Meeting of the European Society for Paediatric Endocrinology (ESPE)
September 10 – 12, 2016 (Paris, France)
M. Beckert, D. Gilfoyle, J.M. Mikkelsen, et al.

Pharmacokinetic Modelling Predicts Native hGH Levels Following Administration of a Sustained-Release Prodrug, TransCon hGH, to Children With GHD
Poster presented at 55th Annual Meeting of the European Society for Paediatric Endocrinology (ESPE)
September 10 – 12, 2016 (Paris, France)
K. Sprogøe, M. Beckert, E.D. Christoffersen, D. Gilfoyle, T. Wegge

TransCon PTH

Sustained Benefit of TransCon PTH, an Investigational Hormone Replacement Therapy for Adult Patients with Hypoparathyroidism, at Week 58 in the PaTH Forward Trial
Late breaking oral presentation at the ASBMR 2021 Annual Meeting
Aliya Khan, Peter Schwarz, Mishaela Rubin, Tamara Vokes, Bart Clarke, Intekhab Ahmed, Andrea Palermo, Claudio Marcocci, Uberto Pagotto, Erik Eriksen, Denka Markova, Susanne Pihl, Michael Beckert, Aimee D. Shu, and Lars Rejnmark

A Single Dose of TransCon PTH in Subjects with Impaired Renal Function: A Phase 1 Trial
Presented at the ASBMR 2021 Annual Meeting
Susanne Pihl , Eshwari Kovoor, Lei Xu, Michael Beckert, and Aimee D. Shu

Week 26 Results from the PaTH Forward Open-Label Extension Trial Support TransCon PTH as a Potential Hormone Replacement Therapy for Patients with Hypoparathyroidism
Presented at ECTS 2021 Digital Congress, April 2021.
L. Rejnmark, P. Schwarz, M. Rubin, T. Vokes, B. Clarke, I. Ahmed, A. Palermo, C. Marcocci, U. Pagotto, E. Eriksen, S. Mourya, D. Markova, S. Pihl, M. Beckert, A. D. Shu, A. Khan

TransCon PTH as a Hormone Replacement Therapy for Patients with Hypoparathyroidism: 6-Month Update from the PaTH Forward Open-Label Extension
Presented at the ENDO 2021, March 2021.
M. Rubin, L. Rejnmark, P. Schwarz, T. Vokes, B. Clarke, I. Ahmed, L. Hofbauer, A. Palermo, C. Marcocci, U. Pagotto, E. Eriksen, S. Mourya, D. Markova, S. Pihl, A.D. Shu, A. Khan.

Disease Burden of Patients Living with Hypoparathyoidism: Results from the Voices of Hypopara Survey.
Presented at the ENDO 2021, March 2021.
D. Murphy, B. Sanders, L. Gulley, A. Knoefler, A. Smith, M. Park, S. Tsao, S. Mourya, M. Rubin.

Design and Topline Results of TransCon PTH, a Long-acting Parathyroid Hormone (PTH), Phase 2 Trial in Patients with Hypoparathyroidism
Presented at the European Calcified Tissue Society (ECTS) 2020 (digital)
October 22 – 24, 2020
D.B. Karpf, S. Mourya, A. Khan, L. Rejnmark

Results of the PaTH Forward Phase 2 Trial Demonstrating Potential of TransCon PTH as a Replacement Therapy for Hypoparathyroidism
Presented at the ASBMR 2020 Virtual Annual Meeting
September 11 – 14, 2020
A. Khan, M. Rubin, L. Rejnmark, P. Schwarz, T. Vokes, B. Clarke, I. Ahmed, L. Hofbauer, A. Palermo, C. Marcocci, U. Pagotto, E. Eriksen, S. Mourya, D. Markova, S. Pihl, D.B. Karpf

Validation of the Hypoparathyroidism Patient Experience Scale (HPES): Understanding and Assessing Burden of Disease
Presented at the ASBMR 2020 Virtual Annual Meeting
September 11 – 14, 2020
M. Brod, D.B. Karpf, A. Smith, J. Gianettoni, D. Markova, S. Mourya, L. McLeod

The Voices of Hypopara Survey: Journey of Patients Living with Hypoparathyroidism
Presented at the ASBMR 2020 Virtual Annual Meeting
September 11 – 14, 2020
D. Astolfi, D. Murphy, B. Sanders, L. Gulley, A. Knoefler, M. Park, S. Tsao, S. Mourya, M. Rubin

TransCon PTH, a Long-acting PTH, in Patients with Hypoparathyroidism: Results of the Phase 2 PaTH Forward Trial
Presented at the e-ECE 2020 virtual meeting
September 5 – 9, 2020
T. Sikjaer, L. Rejnmark, A. Khan, P. Schwarz, T. Vokes, B. Clarke, M. Rubin, L. Hofbauer, E. Eriksen, A. Palermo, U. Pagotto, C. Marcocci, I. Ahmed, S. Mourya, D. Markova, D.B. Karpf

Prevalence of Hypoparathyroidism in the EU: A Systematic Review and Meta-Analysis
Presented at the e-ECE 2020 virtual meeting
September 5 – 9, 2020
D.B. Karpf, C. Catsburg, A. Smith

Design of the PaTH Forward Phase 2Trial ofTransCon PTH, a Long-acting PTH, in Patients with Hypoparathyroidism
Abstract presented at the ENDO Online 2020 virtual meeting
June 8 – 22, 2020
A.A. Khan, L. Rejnmark, P.E. Schwarz, T.J. Vokes, B.L. Clarke, M.R. Rubin, L.C. Hofbauer, Erikson, A. Palermo, U. Pagotto, C. Marcocci, I. Ahmed, S. Mourya, D. Markova, D.B. Karpf

A Randomized Double‐Blind Placebo‐Controlled First‐In‐Human Phase 1 Trial of TransCon PTH in Healthy Adults
Epub
2020 Mar 25
D.B. Karpf, S. Pihl, S. Mourya, E. Mortensen, E. Kovoor, D. Markova, J.A. Leff. J Bone Miner Res. 2020

Design and Preclinical Development of TransCon PTH, an Investigational Sustained-Release PTH Replacement Therapy for Hypoparathyroidism
Epub
October 9, 2019
L. Holten-Andersen, S. Pihl, C.E. Rasmussen, J. Zettler, G. Maitro, J. Baron, S. Heinig, E. Hoffmann, T. Wegge, M. Krusch, F. Faltinger, S. Killian, K. Sprogøe, D. B. Karpf, V. M. Breinholt, F. Cleemann. J Bone Miner Res. 2019

The Effect of TransCon PTH, a Long-acting PTH(1-34), on Serum and Urine Phosphate in Healthy Subjects, and Design of the PaTH Forward Phase 2 Trial
Poster presented at the American Society for Bone and Mineral Research (ASBMR) 2019 Annual Meeting
September 20 – 23, 2019 (Orlando, FL)
B. Karpf, S. Mourya, Z. Lin, E. Kovoor, J. A. Leff

Impacts of Hypoparathyroidism on Patient Work Productivity and Out-of-Pocket Costs
Poster presented the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Annual Meeting
May 18 – 22, 2019 (New Orleans, LA)
L.T. Waldman, M. Brod, A. Smith, D.B. Karp

The Design of a Long-acting PTH (TransCon™ PTH) Phase 2 Trial in Patients with Hypoparathyroidism, Based on Phase 1 Results in Healthy Subjects
Poster presented at the 46th annual meeting of the European Calcified Tissue Society (ECTS)
May 11 – 14, 2019 (Budapest, Hungary)
D.B. Karpf, S. Pihl, S. Mourya, E. Kovoor, J. A. Leff

Understanding the Patient-centered Impact of Hypoparathyroidism on Functioning and Well-being
Poster presented at the Endocrine Society (ENDO) Annual Meeting and Expo
March 23 – 26, 2019 (New Orleans, LA)
M. Brod, L.T. Waldman, A. Smith, D.B. Karpf

TransCon PTH for Hypoparathyroidism—Design and Results of a Phase 1 TransCon PTH Trial in Healthy Volunteers
Poster presented at the American Society for Bone and Mineral Research (ASBMR) 2018 Annual Meeting
September 28 – October 1, 2018 (Montreal, Canada)
D.B Karpf, S. Pihl, E. Mortensen, E. Kovoor, S. Mourya, K. Sprogøe, J.A. Leff

The Effects of TransCon PTH on Bone Turnover Markers in a Phase 1 Trial
Poster presented at the American Society for Bone and Mineral Research (ASBMR) 2018 Annual Meeting
September 28 – October 1, 2018 (Montreal, Canada)
D.B. Karpf, S. Pihl, A. Shu, E. Kovoor, S. Mourya, E. Mortensen, J.A. Leff

TransCon PTH for Hypoparathyroidism – Design and Preliminary Results of a Phase 1 TransCon PTH Trial in Healthy Adults
Poster presented at the 20th European Congress of Endocrinology (ECE)
May 19 – 22, 2018 (Barcelona, Spain)
D.B. Karpf, S. Pihl, E. Mortensen, K. Sprogøe, J. A. Leff

TransCon PTH, a Sustained-Release PTH Prodrug for the Treatment of Hypoparathyroidism: Proof-of-Principle in Cynomolgus Monkeys and TPTx Rats
Poster presented at the American Society for Bone and Mineral Research (ASBMR) 2017 Annual Meeting
September 8 – 11 (Denver, CO)
L. Holten-Andersen, M. Guillot, F. Cleemann, M. Felx, S. Pihl, K. Sprogøe, D. Karpf, A. Valera, V.M. Breinholt

Pharmacokinetics of TransCon PTH, a Sustained-Release PTH Prodrug for Hypoparathyroidism, in Rat and Cynomolgus Monkey
Poster presented at the American Society for Bone and Mineral Research (ASBMR) 2017 Annual Meeting
September 8 – 11 (Denver, CO)
S. Pihl, M. Krusch, L. Holten-Andersen, J. Zettler, F.Cleemann, C. Rasmussen, K. Sprogøe, D. Karpf, V.M. Breinholt

TransCon PTH, a Sustained-Release PTH Analogue for the Treatment of Hypoparathyroidism – Proof-of-Principle in Monkeys and Tptx Rats
Poster presented at the Endocrine Society (ENDO) Annual Meeting and Expo
April 1 – 4, 2017 (Orlando, FL)
K. Sprogøe, L. Holten-Andersen, F. Cleeman, J. Zettler, S. Pihl, V.M. Breinholt

Pharmacokinetics of TransCon PTH, a Sustained-Release PTH Prodrug for Hypoparathyroidism, in Rat and Cynomolgus Monkey
Poster presented at the Endocrine Society (ENDO) Annual Meeting and Expo
April 1 – 4, 2017 (Orlando, FL)
K. Sprogøe, S. Pihl, C.E. Rasmussen, L. Holten-Andersen, F. Cleeman, V.M. Breinholt

TransCon PTH for Hypoparathyroidism – Design and Preliminary Results of a Phase 1 TransCon PTH Trial in Healthy Adults
Poster presented at the Endocrine Society (ENDO) Annual Meeting and Expo
March 17 – 20, 2018 (Chicago, IL)
D.B. Karpf, S. Pihl, E. Mortensen, K. Sprogøe, J.A. Leff

TransCon CNP

Baseline Demographics of the ACHieve Study: A Five-Year, Multi-National Observational Cohort Study of Children with Achondroplasia
Presented at the ASBMR 2021 Annual Meeting
Leanne M. Ward, Ravi Savarirayan, Philippe M. Campeau, Yuri Zarate, Ciara McDonnell, Janet Legare, Daniel Hoernschemeyer, Wolfgang Högler, Dirk Schnabel, Melita Irving, Yongguo Yu, Ying Zhang, Weijian Liu, Dorthe Viuff, Marie-Louise Hartoft-Nielsen Baselin

Design and Baseline Demographics of a Five-Year, Multi-National Observational Cohort Study of Children with Achondroplasia (ACHieve study)
Presented at the ENDO 2021, March 2021.
L.M. Ward, J. Maria de Bergua, M. Robinson, Y. Zhang, C. Decker, A. Giwa, R.W. Charlton.

Design of the ACcomplisH Trial: A Phase 2, Multicenter, Placebo-controlled, Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Weekly TransCon CNP in Children with Achondroplasia
Presented at the ENDO 2021, March 2021.
R. Savarirayan, Y. Zarate, J. Legare, C. Decker, A. Giwa, Y. Zhang, R.W. Charlton, M. Beckert.

Pediatric Achondroplasia: Impacts on Children’s Functioning and Well-being
Presented at the Pediatric Endocrinology Nursing Society (PENS) 2020 national conference (virtual)
November 2 – 5, 2020
K.M. Pfeiffer, M. Brod, D. Bauri, D. Viuff, S. Ota, J. Gianettoni, W. Charlton, A. Smith

Experiences of Parents of Children with Achondroplasia: Impacts on Quality of Life
Presented at the Pediatric Endocrinology Nursing Society (PENS) 2020 national conference (virtual)
November 2 – 5, 2020
K.M. Pfeiffer, M. Brod, D. Bauri, D. Viuff, S. Ota, J. Gianettoni, W. Charlton, A. Smith

Development of a Parent Experience Measure for Parents of Children with Achondroplasia
Poster presented at the 58th Annual European Society for Paediatric Endocrinology Meeting (ESPE)
September 19 – 21, 2019 (Vienna, Austria)
K.M. Pfeiffer, M. Brod, D. Viuff, S. Ota, J. Gianettoni, J.A. Leff

Development of a Measure for the Impacts of Pediatric Achondroplasia on Children’s Daily Functioning and Well-being
Poster presented at the 58th Annual European Society for Paediatric Endocrinology Meeting (ESPE)
September 19 – 21, 2019 (Vienna, Austria)
K.M. Pfeiffer, M. Brod, D. Viuff, S. Ota, J. Gianettoni, J.A. Leff

Experiences of Parents of Children with Achondroplasia: Impacts on Quality of Life
Poster presented at the International Skeletal Dysplasia Society (ISDS) Annual Meeting
September 11 – 14, 2019 (Oslo, Norway)
K.M. Pfeiffer, M. Brod, D. Viuff, S. Ota, J. Gianettoni, J.A. Leff

An Estimate of the Global Birth Prevalence of Achondroplasia
Poster presented at the International Skeletal Dysplasia Society (ISDS) Annual Meeting
September 11 – 14,
2019 (Oslo, Norway)
A. Smith, A. Hill-Ricciuti, S. Anderson, D. Viuff, C. Catsburg, J.A. Leff

Pediatric Achondroplasia: Impacts on Children’s Functioning and Well-being
Poster presented at the
International Skeletal Dysplasia Society (ISDS) Annual Meeting
September 11 – 14, 2019 (Oslo, Norway)
K.M. Pfeiffer, M. Brod, D. Viuff, S. Ota, J. Gianettoni, J.A. Leff

TransCon CNP: A Once Weekly Novel C-type Natriuretic Peptide Therapy in Children with Achondroplasia
Presented at the International Skeletal Dysplasia Society (ISDS) Annual Meeting
September 11 – 14, 2019 (Oslo, Norway)
S. Ota, E.D. Christoffersen, P. Mygind, J. Gianettoni, Z. Lin, D. Viuff, D.B. Karpf, M.
Beckert, V.M. Breinholt, J.A. Leff

TransCon CNP, a Sustained-Release C-Type Natriuretic Peptide Prodrug, a Potentially Safe and Efficacious New Therapeutic Modality for the Treatment of Comorbidities Associated with Fibroblast Growth Factor Receptor 3-Related Skeletal Dysplasias
Epub
June 24, 2019
V.M. Breinholt, C.E. Rasmussen, P.H. Mygind, M. Kjelgaard-Hansen, F. Faltinger, A. Bernhard, J. Zettler, U. Hersel. J Pharmacol Exp Ther. 2019

Structural Optimization of TransCon CNP – Development of a Sustained-Release Prodrug of CNP for Achondroplasia
Poster presented at the 67th Annual Meeting of the American Society of Human Genetics (ASHG)
October 17-21, 2017 (Orlando, FL)
K. Sprogøe, C. Rasmussen, A. Bernhard, F. Faltinger, T. Woods, S. Schröder, A. Brennauer, J. Zettler, T. Wegge, U. Hersel, V.M. Breinholt

TransCon CNP, a Sustained-Release Prodrug of C-type Natriuretic Peptide, Prevents Premature Synchondroses Closure in an Achondroplasia Mouse Model
Poster presented at the American Society for Bone and Mineral Research (ASBMR) 2017 Annual Meeting
September 8 – 11 (Denver, CO)
V.M. Breinholt, M. Cornille, N. Kaci, L. Legeai-Mallet

Pharmacokinetics and Cardiovascular Assessment of TransCon CNP, a Sustained-Release C-type Natriuretic Peptide Prodrug, for the Treatment of Achondroplasia
Poster presented at the American Society for Bone and Mineral Research (ASBMR) 2017 Annual Meeting
September 8 – 11 (Denver, CO)
C.E. Rasmussen, K. Sprogøe, U. Hersel, T. Wegge, F. Faltinger, P.H. Mygind, and V.M. Breinholt

TransCon CNP, a Sustained-Release Prodrug of C-Type Natriuretic Peptide, Exerts Positive Effects on Bone in Juvenile Cynomolgus Monkeys and in a Mouse Model of Achondroplasia
Poster presented at the American Society for Bone and Mineral Research (ASBMR) 2017 Annual Meeting
September 8 – 11 (Denver, CO)
V.M. Breinholt, N. Kaci, C.E. Rasmussen, O. Keil, S. Adermann, U. Hersel, M. Cornille, M. Guillot, N. Doyle, A. Valera; P. Mygind, K. Sprogøe, L. Legeai-Mallet

TransCon CNP, a Sustained-Release Prodrug of C-Type Natriuretic Peptide, Effects Positive Bone Growth in Young Cynomolgus Monkeys and in a Mouse Model of Achondroplasia
Poster presented at the Endocrine Society (ENDO) Annual Meeting and Expo
April 1 – 4, 2017 (Orlando, FL)
V.M. Breinholt, N. Kaci, C.E. Rasmussen, O. Keil, S. Adermann, U. Hersel, M. Cornille, P. H. Mygind, K. Sprogøe, L. Legeai-Mallet

TransCon CNP, a Sustained-Release C-Type Natriuretic Peptide Analogue, for the Treatment of Achondroplasia
Poster presented at the Endocrine Society (ENDO) Annual Meeting and Expo
April 1 – 4, 2017 (Orlando, FL)
K. Sprogøe, C.E. Rasmussen, U. Hersel, V.M. Breinholt

Oncology

Tumor Growth Inhibition Mediated by A Single Dose of Intratumoral TransCon™ TLR7/8 Agonist Associated with Activated Circulating T and B cells and Sustained Low Levels of Systematic Cytokines
Presented at SITC 2021 (Society for Immunotherapy of Cancer) 36th Annual Meeting
Nov 10-14, 2021
Karan Uppal, Luis A. Zuniga, Kathy Bang, Enping Hong, Simran Sabharwal, Yuchi Lee, Salomon Martinez, David Rosen, Amer M. Mirza, Juha Punnonen

A Single Dose of Intratumoral TransCon™ TLR7/8 Agonist Monotherapy Promoted Sustained Activation of Antigen Presenting Cells Resulting in CD4+ and CD8+ T cell Activation and Tumor Growth Inhibition
Presented at SITC 2021 (Society for Immunotherapy of Cancer) 36th Annual Meeting
Nov 10-14, 2021
Luis A. Zuniga, Karan Uppal, Kathy Bang, Enping Hong, Simran Sabharwal, Yuchi Lee, Salomon Martinez, David Rosen, Amer M. Mirza, Juha Punnonen

TransCon™️ IL-2 β/γ: A Novel Long-acting Prodrug of Receptor-biased IL-2 Designed for Improved Pharmocokinetics and Optimal Activation of T Cells for the Treatment of Cancer
Poster presented at the American Association of Cancer Research (AACR) 2020 Virtual Annual Meeting II
June 22 – 24 (virtual)
D.B. Rosen, B. Laufer, T. Knappe, M. Schnabel, D. Begaliew, J. Zettler, M. Krusch, Y. Hehn, A. Bernhard, S. Heinig, V. Konjik, T. Wegge, Y. Wu, W. Song, V. Breinholt, M. Tabrizi, K. Sprogøe, J. Punnonen

Intratumoral Delivery of TransCon™️ TLR7/8 Agonist Provides Potent Anti-tumor Activity as a Monotherapy and in Combination with IL-2 while Minimizing Systemic Cytokine Induction
Poster presented at the 34th Annual Meeting and Pre-Conference Programs for the Society for Immunotherapy of Cancer (SITC)
November 6-10, 2019 (National Harbor, ME)
L.A. Zuniga, T. Leßmann, L. Holten-Andersen, N. Bisek, J. Zettler, S. Stark, F. Faltinger, O. Kracker, S. Weisbrod, R. Müller, T. Voigt, K. Bigott, M. Tabrizifard, V. Breinholt, K. Sprogøe, J. Punnonen

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