نتائج البحث

Natural disease course modeling of achondroplasia to evaluate the efficacy of recifercept in the absence of a placebo control arm in phase II study.

• Authors : Qiao, Wenlian¹ (AUTHOR) ; Boucher, Martin² (AUTHOR); Slade, Alison³ (AUTHOR)

• Source: CPT: Pharmacometrics & Systems Pharmacology. Jul2024, Vol. 13 Issue 7, p1103-1116. 14p.

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Spinal Postures and Mobility in Children with Achondroplasia vs. Age- and Sex-Matched Healthy Individuals: A Preliminary Report.

• Authors : Bayartai, Munkh-Erdene^1,2 (AUTHOR) ; Luomajoki, Hannu¹ (AUTHOR); Aliverti, Andrea³ (AUTHOR)

• Source: Journal of Clinical Medicine. Apr2024, Vol. 13 Issue 7, p2135. 11p.

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Facilitators and constraints of physical activity in adults with achondroplasia: a scoping review.

• Authors : Alves, Inês^1,2 ; Koromani, Fjorda³; Lemos, Carolina^2,4,5

• Source: Journal of Rare Diseases. Dec2024, Vol. 3 Issue 1, p1-23. 23p.

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Growth patterns of young achondroplasia patients in Korea and predictability of neurosurgical procedures.

• Authors : Lee, Jong Seok¹ (AUTHOR); Shim, Youngbo¹ (AUTHOR); Cho, Tae-Joon² (AUTHOR)

• Source: Orphanet Journal of Rare Diseases. 10/5/2023, Vol. 18 Issue 1, p1-11. 11p.

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Achondroplasia with macular coloboma and cone-rod dystrophy: a case report.

• Authors : El-Mofty, Randa M. A. M.¹ ; Hassan, Mai M. I.²

• Source: Journal of the Egyptian Ophthalmological Society. Oct-Dec2023, Vol. 116 Issue 4, p277-280. 4p.

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CORONARY DISEASE AND AORTOPATHY IN ACHONDROPLASIA.

• Authors : DOLAMIĆ, Borislav¹ ; DOLAMIĆ, Ana ALADIN²; STEFANOVIĆ, Maja³

• Source: Medical Review / Medicinski Pregled. sep/okt2023, Vol. 76 Issue 9/10, p295-298. 4p.

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Achondroplasia Natural History Multicenter Clinical Study

• Authors : University of Wisconsin, Madison; Alfred I. duPont Hospital for Children; University of Texas

• Source: Registry of Patients With Bone Disorders

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Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of SAR442501 in Pediatric Participants With Achondroplasia (upreACH-2)

• Source: A Phase 2, Open-label, Multi-center, 2-stage Sequential Cohort, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Subcutaneous SAR442501 in

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A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combination With Lonapegsomatropin in Children With Achondroplasia (COACH)

• Source: A Phase 2, Open-Label, Single-Arm, 156-week Trial to Investigate the Efficacy, Safety and Tolerability of Combined Once Weekly Navepegritide and Lonapegsomatropin in Children With

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A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia (PROPEL3)

• Source: A Phase 3, Multicenter, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Infigratinib in Children 3 to

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