Dedicated to Pitt Hopkins research & treatment, and to creating a network of friends & supporters of those with PTHS. Join us on FB @ https://xitter.cf/t.co/f6wLuyrtXx.
Joined February 2014
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Please join us on November 13 for our virtual EL-PFDD meeting for Pitt Hopkins syndrome!
📅 Friday, November 13, 2026
10 a.m.–2:30 p.m. EST
🔗 Register: us06web.zoom.us/meeting/regi…
📋 Agenda, Family Guide & Meeting Details: pitthopkins.org/pfddmeeting/
#pitthopkins #FDA #ELPFDD
September is #PittHopkins awareness month! Join us in raising awareness all month long – moving research forward, treatments to the clinic and hope for families around the world. pitthopkins.org/donate
Donations doubled during the month of September! #Awareness #RareDisease
📅 Save the Date! November 13, 2026
The Pitt Hopkins Research Foundation is proud to host an Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting with the FDA.
Learn more about the meeting at pitthopkins.org/pfddmeeting.
Registration is Open for the 2026 Pitt Hopkins Virtual Science Symposium!
Registration is Free. Learn more and register today:
📷 pitthopkins.org/2026conferen…
#PittHopkins #PittHopkinsSyndrome #PHRF #RareDisease #Research #ScienceSymposium #PTHS #HopeThroughResearch
We are so excited to join Unravel Biosciences in announcing that today the first person has been dosed in the RVL-001 clinical trial for #PittHopkins syndrome.
pitthopkins.org/unravelannou…
On Wednesday, March 18, Pitt Hopkins families joined Mahzi Therapeutics on #CapitolHill to advocate for change — and it was a powerful day!
We met with lawmakers, shared our stories, and pushed for real progress in rare disease research and regulatory clarity.
#PittHopkins
NEW YORK area families!
The Seaver Autism Center for Research and Treatment at Mount Sinai in New York City is recruiting for a study specific to #PittHopkins.
To find out more, please email: [email protected]
You will be compensated $100 for your time and effort.
Pitt Hopkins Research Foundation retweeted
🎉 A patient w/Pitt-Hopkins syndrome has received an investigational, 1st-in-human gene therapy, thx to the lab of SSCI's Alysson Muotri, PhD. It's the first drug developed using brain organoids — and not animal models — to reach clinical trial. 🧠 H/t @UCSDMedSchool @PittHopkins
Rare Disease Day – February 28, 2026
Rare diseases affect 1 in 10 people worldwide. Most have no approved treatment. How to help:
Donate to fuel research & clinical trials–pitthopkins.org/donate
Participate in research–pitthopkins.org/research/how…
#RareDiseaseDay2026 #PittHopkins
First patient dosed with Mahzi Gene Therapy for Pitt Hopkins - A momentous day for our community!! Read more: pitthopkins.org/mahzipressre…
Children’s Hospital Colorado is now recruiting for the Mahzi gene therapy clinical trial for #PittHopkins syndrome—the third site now open.
📷 Learn more at: pitthopkins.org/mahzi
Neuren shared an FDA update on NNZ-2591 for Pitt Hopkins syndrome (PTHS). The FDA indicated a PTHS-specific clinical measure plus an observer-reported outcome may be appropriate endpoints for a future trial. Steps toward a PTHS study in 2026.
🔗 Read here: neurenpharma.com/showdownloa…
#PittHopkins #Census Count, end of quarter 4 in 2025..
• 1,607 diagnosed with Pitt Hopkins syndrome 💙
• 63 diagnosed with Pitt Hopkins-Like syndrome (1 or 2) 💙
• 77 different countries🌎
• 48 states in the USA🇺🇸
• 0-52 years old🙋♀️🙋
🔗pitthopkins.org/census
#raredisease
We’re excited to share that Rush University Medical Center (Chicago, IL) is now open for enrollment for the Mahzi #genetherapy clinical trial for #PittHopkins syndrome—the second site to begin enrolling participants.
pitthopkins.org/mahzi
📅 July 10–11, 2026 | Science. Hope. Community.
The #PittHopkins Science Symposium is going virtual in 2026, bringing our global community together like never before 🌍💙
🔜 Registration details and agenda will be shared closer to the event dates. pitthopkins.org/2026conferen…
We’re thrilled to announce that Mahzi Therapeutics’ Pitt Hopkins gene therapy clinical trial is opening enrollment at the first site, UCSF Benioff Children’s Hospital Oakland in California!
📷 Learn more at pitthopkins.org/mahzi/.
Updated information now available for the Mahzi Gene Therapy Trial, including
-Community Meeting Recording available to watch
-Updated FAQ section
-Site locations
Check out our website at pitthopkins.org/mahzi for all the latest!
Pitt Hopkins Research Foundation retweeted
September 18th is Pitt Hopkins Awareness Day, so here at Shelby-Rising City we LIT IT UP BLUE in ‘Stile’ for our amazing friend! Huge shout-out to @PittHopkins for the swag! @srchuskies @speakingofstacy
In honor of #PittHopkins Awareness Day, we’ve released a special edition newsletter! Read it now and share with your friends & family: pitthopkins.org/wp-content/u…
Together, we raise awareness. Together, we bring hope.
#PittHopkinsAwarenessDay #PTHS #RareDisease #PHRF #awareness
💙It’s Pitt Hopkins Awareness Day💙
Today we honor the beautiful faces of those touched by Pitt Hopkins Syndrome — the real reason behind every step we take, every dollar raised, and every ounce of hope we hold.
Join us in spreading awareness today! pitthopkins.org
