Tags
- Adrian Bird, PhD 19
- adult care 7
- Alcyone Therapeutics 2
- Angelman syndrome 6
- autism 9
- AveXis 2
- awards 16
- awareness 4
- Baylor College of Medicine 7
- Beam Therapeutics 4
- biomarkers 4
- biopharmaceutical programs 6
- biorepository 7
- biosensors 5
- bone marrow transplant 5
- Boston Children’s Hospital 2
- boys and Rett syndrome 6
- breathing 8
- business development 1
- Case Western Reserve University 4
- CDKL5 2
- Children’s Hospital at Montefiore 8
- cholesterol 7
- chromosomes 1
- clinical initiatives 25
- clinical trials 41
- COVID-19 4
- CRISPR 17
- cystic fibrosis 3
- deep brain stimulation 5
- digital natural history study 3
- DNA 7
- drug delivery 1
- drug discovery 6
- Duchenne muscular dystrophy 2
- events 1
- FDA 10
- FOXG1 2
- Fragile X 9
- Gail Mandel, PhD 20
- gastrointestinal 4
- gene editing 11
- gene replacement 16
- gene therapy 23
- Gene Therapy Consortium 11
- Harvard University 22
- Herophilus 2
- Huda Zoghbi, MD 18
- Jeannie Lee, MD, PhD 5
- ketamine 8
- long genes 6
- MECP2 47
- MECP2 Consortium 21
- MECP2 Duplication Fund 5
- MECP2 duplication syndrome 12
- MECP2 protein 18
- MECP2 reactivation 11
- Michael Greenberg, PhD 13
- Milken Institute 1
- missense mutations 3
- Monica Justice, PhD 12
- Nationwide Children’s Hospital 4
- NIH 9
- Ohio State University 4
- outcome measures 11
- pediatric care 10
- Penn State 2
- protein replacement 5
- related disorders 15
- Rett mice 14
- Rett Syndrome Clinic Atlanta 2
- Rett Syndrome Global Registry 4
- Reverse Rett LA 2
- Reverse Rett NYC 2
- RNA editing 11
- RNA trans-splicing 5
- Roadmap to a Cure 24
- school 6
- scientific initiatives 27
- scoliosis 4
- Shape Therapeutics 3
- siblings 1
- Simons Foundation 3
- stem cells 2
- Taysha Gene Therapies 11
- therapeutics 15
- Translational Medicine Advisory Board 1
- Tri-State Rett Syndrome Center 3
- understanding MECP2 2
- University of Edinburgh 10
- University of Massachusetts School of Medicine 9
- University of North Carolina at Chapel Hill 8
- University of Siena 1
- urology 2
- Vico Therapeutics 4
- Wave Life Sciences 1
- X chromosome 14