HIGHLIGHTS:

 

WE ARE GROWING:

• Establishment of WWOX Europe

• Connected with 30 new patients and their families

• New countries added:  first patients connected with us from Greece, Morocco and Poland

• Added new amazing parent volunteers

 

FUNDRAISING

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AS ONE GLOBAL FOUNDATION WE RAISED OVER $83,250 IN 2024

USA- $34,500

EUROPE  - $25,000 (EUR 23,000)

AUSTRALIA - $23,750 (AUD 37,860)

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Thank you to all our generous donors!

 

RESEARCH

• Our gene therapy pharmaceutical partner (Mahzi Therapeutics) has made tremendous strides and will soon begin manufacturing of the vector to be used in future clinical trials 

• Our academic research partners are working hard to identify the critical functions that the WWOX protein is responsible for in the brain. 

• Establishment of WWOX Patient Sample BioRepository with our partner CombinedBrain

• We awarded the Children’s Medical Research Institute (CMRI) in Sydney, Australia $25,000 (A$40,000) to conduct a small drug-repurposing study.  This study, conducted by Dr. Wendy Gold and Dr. Lisa Riley, will evaluate the potential efficacy of translational readthrough inducing drugs on WWOX nonsense variant read-through. 

 

ADVOCACY

• Connected newly diagnosed families to resources, medical professionals, and other families

• Participated in advocacy and scientific conferences 

 

2025 FOCUS

 

• Roll out of new WWOX Patient Registry

• Continued investigation into drug repurposing options for WWOX patients

• Participation and Funding of research projects to identify WWOX Biomarkers

• Continued Partnership with Mahzi Therapeutics as they continue to progress in development of a WWOX gene therapy and eventual Phase 1 clinical trial