CELL AND GENE THERAPY INSIGHTS

Rare Diseases – Clinical & Commercial Developments

July 2018
Rare Diseases – Clinical & Commercial Developments

Editorial

Unique Role of Patient Advocacy Groups in Clinical Development of CGTx for Rare Diseases

Unique Role of Patient Advocacy Groups in Clinical Development of CGTx for Rare Diseases

Michelle Berg
16 October 2018
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Developing Gene Therapy for CEP290-Associated Retinal Ciliopathies

Developing Gene Therapy for CEP290-Associated Retinal Ciliopathies

Hemant Khanna
2 October 2018
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Expert Insight

Advances & challenges in developing gene therapies for rare kidney diseases

Advances & challenges in developing gene therapies for rare kidney diseases

Valeria Padovano, Michael J Caplan
4 January 2019
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Gene Therapy for Childhood Cerebral Adrenoleukodystrophy

Gene Therapy for Childhood Cerebral Adrenoleukodystrophy

Christine Duncan
17 October 2018
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Gene replacement therapy for hemoglobinopathies: clinical benefit & challenges for widespread utilization

Gene replacement therapy for hemoglobinopathies: clinical benefit & challenges for widespread utilization

M Cavazzana, A Miccio, I Andre-Schmutz et al.
16 October 2018
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Interview

SR-TIGET: Continuing to Drive Enabling Technology Development & Clinical Innovation in Rare Diseases

SR-TIGET: Continuing to Drive Enabling Technology Development & Clinical Innovation in Rare Diseases

Alessandro Aiuti
17 October 2018
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The Critical Role of Patient Advocacy Groups in the Development of Rare Disease Gene Therapies

The Critical Role of Patient Advocacy Groups in the Development of Rare Disease Gene Therapies

Susan Wilson, Elisabeth Linton
16 October 2018
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The Future of Gene Therapy for Rare Eye Diseases

The Future of Gene Therapy for Rare Eye Diseases

Aniz Girach
16 October 2018
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Leukodystrophies: a Patient Advocacy Perspective

Leukodystrophies: a Patient Advocacy Perspective

Maria Kefalas
16 October 2018
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Advancing the Development of AAV-Based Gene Therapy for Rare Diseases

Advancing the Development of AAV-Based Gene Therapy for Rare Diseases

Suyash Prasad
14 October 2018
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