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.
https://t.co/GkxtBsRbiJ
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.
🎉 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–https://t.co/GxBRCuaDew
Participate in research–https://t.co/3Yq9zusX5Y
#RareDiseaseDay2026#PittHopkins
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: https://t.co/JuRfJNpirE
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: https://t.co/HB8kDXWTkl
#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🙋♀️🙋
🔗https://t.co/2YXtqCsEYf
#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.
https://t.co/JuRfJNpirE
📅 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. https://t.co/2Nms7RXaig
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 https://t.co/GHHLryNHbu.
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 https://t.co/Fn1AEqdlP1 for all the latest!
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: https://t.co/fwh6ARhzPh
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! https://t.co/azJrNkEsO9
🌎 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: https://t.co/4TiZyou5lg
#PittHopkins
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
➡ https://t.co/4Hymfs6fYx
THANK YOU, for your continued support of our loved ones and our foundation! 💙
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.
https://t.co/mSLZbLvYZd