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Publication
Financial considerations in expanded access policy for gene therapies: A tough nut to crack?
Molecular Therapy -
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 -
Publication
Facing ethical concerns in the age of precise gene therapy: Outlook on inherited arrhythmias
World Journal of Cardiology -
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
Innovative genoceuticals in human gene therapy solutions: Challenges and safe clinical trials of orphan gene therapy products
Current Gene Therapy -
Publication
Perceptions of airway gene therapy for cystic fibrosis
Expert Opinion on Biological Therapy -
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Publication
Gene therapy: evidence, value and affordability in the US health care system
Journal of Comparative Effectiveness Research -
Publication
Genetic research as therapy: Implications of "gene therapy" for informed consent
J Law Med Ethics