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Adrian Bird, PhD 20

Alcyone Therapeutics 5

Angelman syndrome 6

Baylor College of Medicine 7

Beam Therapeutics 5

Boston Children’s Hospital 3

Brian Kaspar, PhD 1

CURE 360 Webinar 11

Case Western Reserve University 4

Children’s Hospital at Montefiore 8

Duchenne muscular dystrophy 2

Gail Mandel, PhD 22

Gene Therapy Consortium 13

Harvard University 23

Huda Zoghbi, MD 18

Jeannie Lee, MD, PhD 5

MECP2 Consortium 23

MECP2 Duplication Fund 5

MECP2 Editing Consortium 1

MECP2 duplication syndrome 12

MECP2 protein 19

MECP2 reactivation 12

Michael Greenberg, PhD 13

Milken Institute 1

Monica Justice, PhD 12

Nationwide Children’s Hospital 4

Ohio State University 4

RNA trans-splicing 5

Rett Syndrome Clinic Atlanta 2

Rett Syndrome Global Registry 4

Reverse Rett LA 2

Reverse Rett NYC 2

Roadmap to Cures 1

Roadmap to a Cure 25

Shape Therapeutics 5

Simons Foundation 3

Steve Gray, PhD 2

Stuart Cobb, PhD 1

Taysha Gene Therapies 27

The Jackson Laboratory 1

Translational Medicine Advisory Board 1

Tri-State Rett Syndrome Center 3

University of Edinburgh 11

University of Massachusetts School of Medicine 9

University of North Carolina at Chapel Hill 8

University of Siena 1

Vico Therapeutics 6

Wave Life Sciences 3

X chromosome 15

biopharmaceutical programs 7

biorepository 9

bone marrow transplant 5

boys and Rett syndrome 6

business development 1

clinical initiatives 26

clinical trials 60

communication 2

cystic fibrosis 3

deep brain stimulation 5

digital natural history study 6

drug delivery 2

drug discovery 7

gastrointestinal 4

gene editing 13

gene replacement 27

gene therapy 41

missense mutations 3

outcome measures 13

pediatric care 13

protein replacement 4

related disorders 15

scientific initiatives 27

therapeutics 15

understanding MECP2 2

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Roadmap to Cures

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