BioInsights - Rare Diseases – Clinical & Commercial Developments - spotlight

CELL & GENE THERAPY INSIGHTS

Rare Diseases – Clinical & Commercial Developments

Guest Editor:

Jaap Boelens, Pediatrician-Oncologist, Immunologist at University Medical Centre Utrecht

July 2018

Advances & challenges in developing gene therapies for rare kidney diseases

V Padovano,

M Caplan

4 January 2019

Expert Insight

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Gene Therapy for Childhood Cerebral Adrenoleukodystrophy

C Duncan

17 October 2018

Expert Insight

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

A Aiuti

17 October 2018

Interview

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

S Wilson,

E Linton

16 October 2018

Interview

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

M Berg

16 October 2018

Editorial

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The Future of Gene Therapy for Rare Eye Diseases

A Girach

16 October 2018

Interview

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Leukodystrophies: a Patient Advocacy Perspective

M Kefalas

16 October 2018

Interview

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

M Cavazzana,

A Miccio,

I Andre-Schmutz et al.

16 October 2018

Expert Insight

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

S Prasad

14 October 2018

Interview

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Developing Gene Therapy for CEP290-Associated Retinal Ciliopathies

H Khanna

2 October 2018

Editorial

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