Section 6 of 6
References
96 sources
- Acikgoz et al. (2026) (opens in a new tab)
- Al Mukaddam et al. (2022) (opens in a new tab)
- Atebrioz Prescribing Information (2026) (opens in a new tab)
- Baujat et al. (2017) (opens in a new tab)
- Baujat et al. (2026) (opens in a new tab)
- BioPharma Dive (2023) (opens in a new tab)
- BioPharma Dive (2026) (opens in a new tab)
- BioSpace (2026) (opens in a new tab)
- Bose et al. (2025) (opens in a new tab)
- Botman et al. (2021) (opens in a new tab)
- Cacco et al. (2025) (opens in a new tab)
- CADTH Pharmacoeconomic Review (2023) (opens in a new tab)
- CADTH Pharmacoeconomic Review (2023) (opens in a new tab)
- CADTH Pharmacoeconomic Review (2023) (opens in a new tab)
- CADTH Reimbursement Recommendation (2023) (opens in a new tab)
- Chen et al. (2020) (opens in a new tab)
- ClinicalTrials.gov NCT04307953 (opens in a new tab)
- ClinicalTrials.gov NCT04455841 (opens in a new tab)
- ClinicalTrials.gov NCT04455841 (opens in a new tab)
- ClinicalTrials.gov NCT04582539 (opens in a new tab)
- ClinicalTrials.gov NCT04582539 (opens in a new tab)
- ClinicalTrials.gov NCT05039515 (opens in a new tab)
- ClinicalTrials.gov NCT05090891 (opens in a new tab)
- ClinicalTrials.gov NCT05099445 (opens in a new tab)
- ClinicalTrials.gov NCT05394116 (opens in a new tab)
- ClinicalTrials.gov NCT06089616 (opens in a new tab)
- ClinicalTrials.gov NCT06508021 (opens in a new tab)
- Di Rocco et al. (2023) (opens in a new tab)
- European Medicines Agency (2023) (opens in a new tab)
- European Medicines Agency (2025) (opens in a new tab)
- FDA (2026) (opens in a new tab)
- FDA Novel Drug Approvals (2026) (opens in a new tab)
- Fierce Pharma (2026) (opens in a new tab)
- Gaechter et al. (2026) (opens in a new tab)
- Harrak et al. (2025) (opens in a new tab)
- Hatsell et al. (2015) (opens in a new tab)
- Hino et al. (2015) (opens in a new tab)
- Hsiao and Pacifici (2025) (opens in a new tab)
- Hsiao et al. (2019) (opens in a new tab)
- IFOPA (opens in a new tab)
- IFOPA Mirum FAQs (2026) (opens in a new tab)
- IFOPA zilurgisertib announcement (2026) (opens in a new tab)
- Incyte Corporation INCB 00928-104 protocol (2025) (opens in a new tab)
- Incyte Corporation INCB 00928-104 statistical analysis plan (2025) (opens in a new tab)
- Incyte Corporation INCB 00928-105 statistical analysis plan (2024) (opens in a new tab)
- Incyte Form 10-K (2026) (opens in a new tab)
- Incyte Form 10-Q (2026) (opens in a new tab)
- International Clinical Council on FOP (2025) (opens in a new tab)
- International Clinical Council on FOP classes of medications (2024) (opens in a new tab)
- International Clinical Council on FOP current treatment considerations (2024) (opens in a new tab)
- International Clinical Council on FOP executive summary (2025) (opens in a new tab)
- Ipsen (2025) (opens in a new tab)
- Ipsen (2026) (opens in a new tab)
- Kaplan et al. (2010) (opens in a new tab)
- Kaplan et al. (2018) (opens in a new tab)
- Kaplan et al. (2025a) (opens in a new tab)
- Kaplan et al. (2025b) (opens in a new tab)
- Keen et al. (2024) (opens in a new tab)
- Kitterman et al. (2005) (opens in a new tab)
- MedCity News (2026) (opens in a new tab)
- Mirum Access Plus (2026) (opens in a new tab)
- Mirum Pharmaceuticals (2026) (opens in a new tab)
- Mirum Pharmaceuticals first-quarter 2026 results (2026) (opens in a new tab)
- Mirum Pharmaceuticals Form 10-Q (2026) (opens in a new tab)
- Mirum Pharmaceuticals PROGRESS results press release (2026) (opens in a new tab)
- Mohan et al. (2023) (opens in a new tab)
- NICE (2025) (opens in a new tab)
- NIHR Innovation Observatory (2025) (opens in a new tab)
- Ortiz-Agapito et al. (2015) (opens in a new tab)
- Pasatru Prescribing Information (2026) (opens in a new tab)
- Peng et al. (2019) (opens in a new tab)
- Pignolo and Kaplan (2018) (opens in a new tab)
- Pignolo et al. (2016) (opens in a new tab)
- Pignolo et al. (2019) (opens in a new tab)
- Pignolo et al. (2020) (opens in a new tab)
- Pignolo et al. (2021) (opens in a new tab)
- Pignolo et al. (2022a) (opens in a new tab)
- Pignolo et al. (2022b) (opens in a new tab)
- Pignolo et al. (2023a) (opens in a new tab)
- Pignolo et al. (2023b) (opens in a new tab)
- Pignolo et al. (2026a) (opens in a new tab)
- Pignolo et al. (2026b) (opens in a new tab)
- Pignolo et al. (2026c) (opens in a new tab)
- Reuters (2026) (opens in a new tab)
- Shore et al. (2006) (opens in a new tab)
- Smilde et al. (2022) (opens in a new tab)
- Sohonos Prescribing Information (2026) (opens in a new tab)
- Swain (2026) (opens in a new tab)
- Ullrich et al. (2025) (opens in a new tab)
- Xu et al. (2025) (opens in a new tab)
- Xun et al. (2022a) (opens in a new tab)
- Xun et al. (2022b) (opens in a new tab)
- Yang et al. (2025a) (opens in a new tab)
- Yang et al. (2025b) (opens in a new tab)
- Yang et al. (2026a) (opens in a new tab)
- Yang et al. (2026b) (opens in a new tab)