Search Results - "Dahmani-Rabehi B"

Evidence of Oxytosis/Ferroptosis in Niemann–Pick Disease Type C.

Authors: Sanchez, Kayla L. Kim, Jeanyoung White, Jacob B. et al.

Source: International Journal of Molecular Sciences. Apr2025, Vol. 26 Issue 7, p2915. 13p.

Clinical characteristics and treatment outcomes in patients with Niemann–Pick disease type C (NP-C): a cross-sectional study.

Authors: Karimzadeh, Parvaneh Ahmadabadi, Farzad Zeinali, Vahide et al.

Source: Orphanet Journal of Rare Diseases. 8/27/2025, Vol. 20 Issue 1, p1-8. 8p.

Exploration of Bromodomain Proteins as Drug Targets for Niemann–Pick Type C Disease.

Authors: Parente, Martina Barthelemy, Amélie Tonini, Claudia et al.

Source: International Journal of Molecular Sciences. Jun2025, Vol. 26 Issue 12, p5769. 22p.

Plasmid Gene Therapy for Monogenic Disorders: Challenges and Perspectives.

Authors: Luís, Marco A. Goes, Marcelo A. D. Santos, Fátima Milhano et al.

Source: Pharmaceutics. Jan2025, Vol. 17 Issue 1, p104. 28p.

Current and Emerging Therapies for Lysosomal Storage Disorders.

Authors: Abelleyra Lastoria DA Keynes S Hughes D

Source: Drugs [Drugs] 2025 Feb; Vol. 85 (2), pp. 171-192. Date of Electronic Publication: 2025 Jan 18.

Publication Type: Journal Article; Review

Journal Info: Publisher: Adis, Springer International Country of Publication: New Zealand NLM ID: 7600076 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1179-1950 (Electronic) Linking ISSN: 00126667 NLM ISO Abbreviation: Drugs Subsets: MEDLINE

Effects of miglustat therapy on neurological disorder and survival in early-infantile Niemann-Pick disease type C: a national French retrospective study.

Authors: Freihuber C Dahmani-Rabehi B Brassier A et al.

Source: Orphanet journal of rare diseases [Orphanet J Rare Dis] 2023 Jul 21; Vol. 18 (1), pp. 204. Date of Electronic Publication: 2023 Jul 21.

Publication Type: Observational Study; Journal Article

Journal Info: Publisher: BioMed Central Country of Publication: England NLM ID: 101266602 Publication Model: Electronic Cited Medium: Internet ISSN: 1750-1172 (Electronic) Linking ISSN: 17501172 NLM ISO Abbreviation: Orphanet J Rare Dis Subsets: MEDLINE

Overview of clinical, molecular, and therapeutic features of Niemann-Pick disease (types A, B, and C): Focus on therapeutic approaches.

Authors: Hosseini K Fallahi J Razban V et al.

Source: Cell biochemistry and function [Cell Biochem Funct] 2024 Jun; Vol. 42 (4), pp. e4028.

Publication Type: Journal Article; Review

Journal Info: Publisher: Wiley-Blackwell Country of Publication: England NLM ID: 8305874 Publication Model: Print Cited Medium: Internet ISSN: 1099-0844 (Electronic) Linking ISSN: 02636484 NLM ISO Abbreviation: Cell Biochem Funct Subsets: MEDLINE

Splenomegaly and progressive neurologic involvement: Think about Niemann-Pick type C disease.

Authors: Yoldas Celik M Yazici H Erdem F et al.

Source: Pediatrics international : official journal of the Japan Pediatric Society [Pediatr Int] 2024 Jan-Dec; Vol. 66 (1), pp. e15832.

Publication Type: Journal Article

Journal Info: Publisher: Blackwell Science Asia Country of Publication: Australia NLM ID: 100886002 Publication Model: Print Cited Medium: Internet ISSN: 1442-200X (Electronic) Linking ISSN: 13288067 NLM ISO Abbreviation: Pediatr Int Subsets: MEDLINE