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PHRF2026-08-14 20:41:372026-08-14 20:41:37PHRF August 2026 NewsletterGene Therapy Clinical Trial: Information now available from Mahzi Therapeutics
Our Mission
The mission of the Pitt Hopkins Research Foundation (PHRF) is to support research dedicated to finding a treatment, and an eventual cure for Pitt Hopkins Syndrome and other similar disorders. The PHRF is also dedicated to supporting the Pitt Hopkins community with resource recommendations, parental support and the latest medical information.
From Lab to Clinic, we’re driving trials forward with unwavering passion!
The California Institute for Regenerative Medicine has awarded a $4 MILLION DOLLAR GRANT to Mahzi Therapeutics to accelerate their work in Pitt Hopkins Gene Therapy.
Latest News
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PHRF2026-08-14 20:41:372026-08-14 20:41:37PHRF August 2026 Newsletter
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PHRF2026-07-21 22:24:042026-09-24 21:40:14Pitt Hopkins Externally-Led Patient-Focused Drug Development Meeting
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PHRF2026-05-12 21:26:572026-05-12 21:28:03Unravel Announces First Patient Dosed in RVL-001 TrialUpcoming Events
Join us for an Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting, where individuals with Pitt Hopkins syndrome, families, caregivers, researchers, industry, and the FDA come together to share lived experiences and discuss the greatest unmet needs to help guide future treatment and drug development.“Because of the joy of giving, we can make a difference for Pitt Hopkins and Autism families. With Pitt Hopkins being a type of Autism, this research can give clues to finding a cure for Autism too.”
“I see how difficult it can be having a special needs child. If my giving a little bit can help give hope to even a tiny portion of the families with special needs children, I am more than happy to do so.”
“I’m simply amazed by what this group of parents (and scientist supporters) has been able to do over the years. Raise awareness, Raise Money!”
“I fundraise because my daughter cannot. I speak up because my daughter cannot. I will continue to fight until “cannot” is not in our vocabulary.”
Upcoming Events
OVER 94% OF ALL FUNDS DONATED GO TO RESEARCH
Our board and officers are parents and professionals who work diligently to ensure over 94% of all funds donated go toward research and our mission of finding a cure.
Latest News
PHRF August 2026 NewsletterAugust 14, 2026 - 8:41 pm
Pitt Hopkins Externally-Led Patient-Focused Drug Development MeetingJuly 21, 2026 - 10:24 pm
Unravel Announces First Patient Dosed in RVL-001 TrialMay 12, 2026 - 9:26 pm
First Patient Dosed with Mahzi Gene TherapyFebruary 25, 2026 - 6:18 pm
