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Updated November 28, 2023 FDA Clinical Investigator Training Course (CITC) 2023: December 6-7 2023 ABOUT THIS EVENT This course is...

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Updated October 15, 2023 Research technician DCRT: Would you like to develop genetic therapies for patients with rare neurological...

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‘This is not a science problem anymore': Paths emerge for scaling up rare disease medicine

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The Cost of Delayed Diagnosis in Rare Disease: A Health Economic Study

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All of Us and the Promise of Precision Medicine:

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MAKING GENETIC THERAPIES AFFORDABLE AND ACCESSIBLE

Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N=1 Exon Skipping ASOs

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Regulatory Guidances

Clinical Recommendations Draft Guidance

IND Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases:...

Regulatory Guidances

CMC Recommendations Draft Guidance

Investigational New Drug Application Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating...

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Nonclinical Testing Draft Guidance

Nonclinical Testing of Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases...

Regulatory Guidances

Administrative and Procedural Draft Guidance

IND Submissions for Individualized Antisense Oligonucleotide Drug Products: Administrative and Procedural Recommendations Guidance for...

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