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Publication
Are we prepared to deliver gene-targeted therapies for rare diseases?
American Journal of Medical Genetics Part C: Seminars in Medical Genetics -
Publication
Financial considerations in expanded access policy for gene therapies: A tough nut to crack?
Molecular Therapy -
Publication
Innovative insurance to improve U.S. patient access to cell and gene therapy
Millbank Quarterly -
Publication
“Gene therapy”: Ethical and regulatory issues
Novel Platforms for Drug Delivery Applications -
Publication
European Pharmacopoeia's approach to cell and gene therapy: Focus on how gene therapy texts are evolving
Springer Cham -
News
Novel Gene Therapies for Pediatric and Young Adults with Sickle Cell Disease: Engaging Patient and Parent Stakeholders Working Group
Sickle Cell Disease (SCD) is an inherited disorder that results in premature mortality and severe morbidity in millions of afflicted individuals worldwide. Novel therapies are urgently needed and…- working group
- gene therapy
- Sickle Cell
- Stakeholder Engagement
- Spotlight
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Publication
Facing ethical concerns in the age of precise gene therapy: Outlook on inherited arrhythmias
World Journal of Cardiology -
Publication
Innovative genoceuticals in human gene therapy solutions: Challenges and safe clinical trials of orphan gene therapy products
Current Gene Therapy -
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Publication
Perceptions of airway gene therapy for cystic fibrosis
Expert Opinion on Biological Therapy