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Rare Diseases
1:00
Trials evaluating the efficacy of CD20 CAR-T cell therapy in WM and CNS lymphoma
Mazyar Shadman
• 9 Dec 2022
2:51
Treating otoferlin deficiency with gene therapy
Nawal Ouzren
• 26 Apr 2022
2:05
Novel gene therapy solutions for Usher syndrome type I
Nawal Ouzren
• 26 Apr 2022
1:59
Developing gene therapies for GJB2 gene related hearing loss
Nawal Ouzren
• 26 Apr 2022
1:38
Overcoming manufacturing issues in gene therapies for rare diseases
John Maslowski
• 27 Jan 2022
2:12
Innovations in treating rare diseases with gene therapies
John Maslowski
• 27 Jan 2022
1:29
OTL-103: An autologous gene therapy for Wiskott Aldrich syndrome
Kent Christopherson
• 27 Jan 2022
2:31
Novel gene therapies for metachromatic leukodystrophy
Kent Christopherson
• 27 Jan 2022
4:51
Challenges in manufacturing viral vectors for the clinic
Nathalie Clement
• 27 Jan 2022
1:58
Treating Krabbe disease with FBX-101
John Maslowski
• 27 Jan 2022
1:06
Increasing patient recruitment to rare disease trials
Will Chou
• 26 Jan 2022
1:57
MOMENTUM: ARU-1801 in sickle cell disease
Will Chou
• 26 Jan 2022
2:26
ARU-1801, a novel lentiviral gene therapy for SCD
Will Chou
• 27 Jan 2022
1:10
Addressing issues in conducting clinical trials for rare diseases
Will Chou
• 27 Jan 2022
1:44
Key developments in the future of advanced therapies
Lavakumar Karyampudi
• 27 Jan 2022
2:23
DSG3-CAART: A novel CAAR T-cell therapy in pemphigus vulgaris
Samik Basu
• 26 Jan 2022
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