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Open access

Research article

First published 2022

Future of genetic therapies for rare genetic diseases: what to expect for the next 15 years?

Luiza Amara Maciel Braga, Carlos Gilbert Conte Filho, Fabio Batista Mota

Therapeutic Advances in Rare Disease

Open access

Review article

First published 2018-12-20

Regenerative Medicine Therapies for Rare Diseases

Larissa Lapteva, Ramjay Vatsan, Tejashri Purohit-Sheth

Translational Science of Rare Diseases

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 2023-09

Advances and Challenges in the Development of Gene Therapy Medicinal Products for Rare Diseases

Juan A. Bueren, Alberto Auricchio

Human Gene Therapy

Retricted access

Review article

First published 2019-10

A New Era for Rare Genetic Diseases: Messenger RNA Therapy

Paolo G.V. Martini, Lin T. Guey

Human Gene Therapy

Retricted access

Article commentary

First published 2013-04

Gene Therapy for Rare Diseases: Summary of a National Institutes of Health Workshop,September 13,2012

Marina O'Reilly, Donald B. Kohn, Jeffrey Bartlett [...]

Human Gene Therapy

Open access

Review article

First published 2018

Improving market access to rare disease therapies: A worldwide perspective with recommendations to the industry

François Lucas

The Journal of Medicine Access

Open access

Research article

First published 2019

Functional rare variants influence the clinical response to anti-TNF therapy in Crohn’s disease

María Chaparro, Adrià Aterido, Iván Guerra [...]

Therapeutic Advances in Gastroenterology

Retricted access

Research article

First published

Improving Gene Therapy Development for Rare Diseases

Larissa Warneck-Silvestrin

Inside Precision Medicine

Open access

Research article

First published 2023-03

Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Experience

Richa Madan Lomash, Oleg Shchelochkov, Randy J. Chandler [...]

Human Gene Therapy

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