Dedicated to Pitt Hopkins research & treatment, and to creating a network of friends & supporters of those with PTHS. Join us on FB @ pitthopkins.org.
Joined February 2014
- Tweets 1,449
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442 Photos and videos
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
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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…
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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
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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: stella.chukwulozie@mssm.edu
You will be compensated $100 for your time and effort.
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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
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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
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First patient dosed with Mahzi Gene Therapy for Pitt Hopkins - A momentous day for our community!! Read more: pitthopkins.org/mahzipressre…
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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
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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…
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#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
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15 Dec 2025
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
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14 Dec 2025
📅 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…
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2 Dec 2025
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/.
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24 Nov 2025
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!
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Pitt Hopkins Research Foundation retweeted
19 Sep 2025
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
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18 Sep 2025
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
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18 Sep 2025
💙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
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16 Sep 2025
🌎 Big News from Brazil!
We are thrilled to share that the Brazilian Association of Pitt-Hopkins Syndrome has officially launched! 🇧🇷
We are excited to collaborate and work together to advance research and advocacy worldwide. Learn more: pitthopkins.org.br
#PittHopkins
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12 Sep 2025
Research is moving forward, gene therapy and clinical trials are happening NOW to help those living with #PittHopkins syndrome. Read our updated pipeline and #research info
➡ pitthopkins.org/pipeline/
THANK YOU, for your continued support of our loved ones and our foundation! 💙
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11 Sep 2025
New Research Alert for the Pitt Hopkins Community!
A new study found that people with #PittHopkins Syndrome may be more likely to have eosinophilic esophagitis (EoE) & issues with the immune system like low antibody levels and frequent infections.
pitthopkins.org/research/res…
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