Samantha Parker
Neuilly-sur-seine, France
Last Updated: 09/26/2026
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Found 15 publications
Non-oncologic orphan drug approvals across the world: Types of evidence required and time to approval.
Journal: Drug discovery today
Published: August 14, 2025
Non-oncology orphan drug development: Productivity and probability of success.
Journal: Drug discovery today
Published: August 08, 2025
Fostering the international interoperability of clinical research networks to tackle undiagnosed and under-researched rare diseases.
Journal: Frontiers in medicine
Published: April 11, 2024
Generalized pairwise comparisons of prioritized outcomes are a powerful and patient-centric analysis of multi-domain scores.
Journal: Orphanet journal of rare diseases
Published: May 31, 2023
Understanding disease symptoms and impacts and producing qualitatively-derived severity stages for MPS IIIA: a mixed methods approach.
Journal: Orphanet journal of rare diseases
Published: September 13, 2021
An observational, prospective, multicenter, natural history study of patients with mucopolysaccharidosis type IIIA.
Journal: Molecular genetics and metabolism
Published: September 10, 2021
Caregivers' assessment of meaningful and relevant clinical outcome assessments for Sanfilippo syndrome.
Journal: Journal of patient-reported outcomes
Published: June 28, 2021
Parent Experiences of Sanfilippo Syndrome Impact and Unmet Treatment Needs: A Qualitative Assessment.
Journal: Neurology and therapy
Published: October 14, 2020
International Cooperation to Enable the Diagnosis of All Rare Genetic Diseases.
Journal: American journal of human genetics
Published: May 06, 2017
Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III.
Journal: Orphanet journal of rare diseases
Published: March 29, 2017
Adeno-Associated Virus-Based Gene Therapy for CNS Diseases.
Journal: Human gene therapy
Published: June 09, 2016
Last Updated: 09/26/2026