Tags
- Adrian Bird, PhD 20
- adult care 8
- Alcyone Therapeutics 5
- Angelman syndrome 6
- autism 9
- AveXis 2
- awards 17
- awareness 4
- Baylor College of Medicine 7
- Beam Therapeutics 5
- biomarkers 6
- biopharmaceutical programs 7
- biorepository 9
- biosensors 9
- bone marrow transplant 5
- Boston Children’s Hospital 3
- boys and Rett syndrome 6
- breathing 9
- Brian Kaspar, PhD 1
- business development 1
- Case Western Reserve University 4
- CDKL5 2
- Children’s Hospital at Montefiore 8
- cholesterol 7
- chromosomes 1
- clinical initiatives 28
- clinical trials 54
- communication 2
- COVID-19 4
- CRISPR 19
- CURE 360 Webinar 11
- cystic fibrosis 3
- deep brain stimulation 5
- digital natural history study 6
- DNA 7
- drug delivery 2
- drug discovery 7
- Duchenne muscular dystrophy 2
- events 6
- FDA 11
- FOXG1 3
- Fragile X 10
- Gail Mandel, PhD 22
- gastrointestinal 4
- gene editing 14
- gene replacement 28
- gene therapy 33
- Gene Therapy Consortium 13
- Harvard University 23
- Herophilus 3
- Huda Zoghbi, MD 18
- Jeannie Lee, MD, PhD 5
- ketamine 8
- long genes 6
- MECP2 48
- MECP2 Consortium 23
- MECP2 Duplication Fund 5
- MECP2 duplication syndrome 12
- MECP2 protein 19
- MECP2 reactivation 13
- Michael Greenberg, PhD 13
- Milken Institute 1
- missense mutations 3
- Monica Justice, PhD 12
- Nationwide Children’s Hospital 4
- Neurogene 8
- NIH 9
- Ohio State University 4
- outcome measures 13
- pediatric care 13
- Penn State 2
- protein replacement 5
- related disorders 15
- Rett mice 17
- Rett Syndrome Clinic Atlanta 2
- Rett Syndrome Global Registry 4
- Reverse Rett LA 2
- Reverse Rett NYC 2
- RNA editing 15
- RNA trans-splicing 6
- Roadmap to a Cure 24
- school 6
- scientific initiatives 28
- scoliosis 4
- Shape Therapeutics 5
- siblings 4
- Simons Foundation 3
- stem cells 2
- Steve Gray, PhD 2
- Stuart Cobb, PhD 1
- Taysha Gene Therapies 21
- The Jackson Laboratory 1
- therapeutics 15
- Translational Medicine Advisory Board 1
- Tri-State Rett Syndrome Center 3
- understanding MECP2 2
- University of Edinburgh 11
- University of Massachusetts School of Medicine 9
- University of North Carolina at Chapel Hill 8
- University of Siena 1
- urology 2
- Vico Therapeutics 6
- Wave Life Sciences 3
- X chromosome 15