Sage Journals: Discover world-class research

Free access

Review article

First published 2018-11

Pharmacovigilance of medicines for rare and ultrarare diseases

Marco Sardella, Glyn Belcher

Therapeutic Advances in Drug Safety

Retricted access

Research article

First published 2025-4

Ultrarare Cause of Childhood Chorea: Celiac Disease

Pinar Ozbudak

Journal of Child Neurology

Retricted access

Research article

First published 2021-3

Treating Ultrarare Diseases,One Patient at a Time

Julianna LeMieux

Inside Precision Medicine

Retricted access

Review article

First published 2023-09

Realizing the Potential of Gene Therapies for Rare and Ultra-Rare Inherited Diseases

Claire Booth, Alessandro Aiuti

Human Gene Therapy

Retricted access

Review article

First published 2022-06

Modulation of RNA Splicing by Oligonucleotides: Mechanisms of Action and Therapeutic Implications

Olga V. Sergeeva, Evgeniya Y. Shcherbinina, Noam Shomron [...]

Nucleic Acid Therapeutics

Open access

Research article

First published 2022-04

Preparing n-of-1 Antisense Oligonucleotide Treatments for Rare Neurological Diseases in Europe: Genetic,Regulatory,and Ethical Perspectives

Matthis Synofzik, Willeke M.C van Roon-Mom, Georg Marckmann [...]

Nucleic Acid Therapeutics

Open access

Research article

First published 2025-10

Rare genetic variants in PKD1 and SMAD2 are associated with intracranial aneurysms in the general population

Bibi M Wolters, Mark K Bakker, Kristiina Rannikmäe [...]

International Journal of Stroke

Retricted access

Review article

First published 2026-5

Ethical and Regulatory Considerations for Developing Gene Therapies Involving Genome Editing

Pishoy Gouda, Lauren Cohen, Keith Berelowitz [...]

Human Gene Therapy

Retricted access

Research article

First published 2026

Novel Homozygous TUBGCP6 Variant Impairs Brain Development: Case Report and Literature Review

Silvia Lelli, Giulia Balletto, Isabelle Bacchi [...]

Journal of Child Neurology

Open access

Research article

First published 2023-04

Translational Pharmacokinetic/Pharmacodynamic Model for mRNA-3927,an Investigational Therapeutic for the Treatment of Propionic Acidemia

Husain Attarwala, Matthew Lumley, Min Liang [...]

Nucleic Acid Therapeutics

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