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ZC4H2 Foundation
Boca Raton, Florida, United States
- Ownership
- Private
- Development stage
- Other
01
Overview
The ZC4H2 Foundation is a non-profit organization dedicated to advancing research and finding treatments for ZC4H2-associated rare disorders (ZARD). ZARD is a group of ultra-rare genetic conditions characterized by neuromuscular symptoms, joint contractures, and developmental delays. The foundation focuses on building a patient registry, funding scientific research, and fostering collaborations between clinicians and researchers globally. Their primary objective is to accelerate the development of therapeutic interventions, including gene-based therapies and small molecule treatments. By raising awareness and providing support to affected families, the foundation serves as a central hub for the ZARD community.
- Therapeutic areas
- Rare Diseases
- Modalities
- Gene therapyBiologicAntisense oligonucleotideSmall molecule
- Industry
- Academic Hospital/University/Research
02
Partnerships
The Jackson LaboratoryUniversity of Pennsylvania
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