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Frontiers
Mastocytosis
Frontiers
RDDC
Orphanet Journal of Rare Diseases | French Guidelines for the Management of Nonadvanced Mastocytosis in Adults
November 07, 2025
Achondroplasia
Hypochondroplasia
Epidemiological Analysis
FGFR3 Gene Mutation
Early Diagnosis
Frontiers
RDDC
Orphanet Journal of Rare Diseases | Nationwide Epidemiological Analysis of Achondroplasia and Hypochondroplasia in France
November 05, 2025
AI
NF1-associated Glioma
Radiomics
Data Scarcity
Frontiers
RDDC
Orphanet Journal of Rare Diseases | Artificial Intelligence Applications in NF1-Associated Gliomas
November 03, 2025
iPSC-derived retinal organoids
inherited retinal diseases
disease modeling
precision therapy
Frontiers
RDDC
Orphanet Journal of Rare Diseases | Advances of iPSC-Derived Retinal Organoids in Inherited Retinal Disease Research
November 02, 2025
Odontogenic Cysts
Microsurgical Treatment
Recurrence Analysis
Gorlin-Goltz Syndrome
Frontiers
RDDC
Orphanet Journal of Rare Diseases | Treatment and Recurrence Analysis of Peripheral Jaw Cysts
November 01, 2025
MEA Matrix
Orphan Drug Reimbursement
Clinical Financial Risks
Systematic Review
Frontiers
RDDC
Orphanet Journal of Rare Diseases | Development of a Managed Entry Agreement Matrix to Address Reimbursement Challenges for Orphan Medicinal Products
October 30, 2025
Gaucher disease
Diagnostic guidelines
Biomarkers
Frontiers
RDDC
Orphanet Journal of Rare Diseases | Update on French National Guidelines for Gaucher Disease Diagnosis and Treatment
October 01, 2025
Laronidase
MPS I
Phase IV
Safety assessment
Frontiers
RDDC
Orphanet Journal of Rare Diseases | Long Ruilin et al. Safety and efficacy of Laronidase in Chinese MPS I patients: A phase IV single-arm open-label multicenter study
April 05, 2025
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