RETT SYNDROME
Clinical trials for RETT SYNDROME explained in plain language.
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One-Time brain injection aims to halt devastating genetic disorder in girls
Disease control Recruiting nowThis study is testing a one-time gene therapy called NGN-401 for girls with Rett syndrome, a severe genetic disorder that impairs brain development. The therapy delivers a working copy of a missing gene directly to the brain. Researchers will follow participants for 3 years to se…
Matched conditions: RETT SYNDROME
Phase: PHASE3 • Sponsor: Neurogene Inc. • Aim: Disease control
Last updated Mar 31, 2026 12:11 UTC
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One-Shot gene therapy trial offers hope for devastating neurological disorder
Disease control Recruiting nowThis study is testing a single injection of an experimental gene therapy called TSHA-102 for females with Rett syndrome. The goal is to see if the treatment is safe and can help improve developmental skills by targeting the genetic root cause of the disease. The study will follow…
Matched conditions: RETT SYNDROME
Phase: PHASE3 • Sponsor: Taysha Gene Therapies, Inc. • Aim: Disease control
Last updated Mar 27, 2026 12:40 UTC
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Virtual therapy aims to ease behavioral challenges in rare genetic disorders
Symptom relief Recruiting nowThis study is testing whether a telehealth therapy program can help children with rare genetic syndromes improve their communication and reduce challenging behaviors. Children aged 2-12 with conditions like Angelman syndrome or Fragile X will work with therapists and their parent…
Matched conditions: RETT SYNDROME
Phase: NA • Sponsor: Rush University Medical Center • Aim: Symptom relief
Last updated Mar 27, 2026 12:38 UTC
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Scientists search for brain clues in rare genetic disorder
Knowledge-focused Recruiting nowThis study aims to identify brain activity patterns that could serve as markers for Rett Syndrome, a rare genetic disorder affecting brain development. Researchers will measure electrical brain signals using EEG tests in girls with Rett Syndrome and compare them to typically deve…
Matched conditions: RETT SYNDROME
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Apr 01, 2026 14:40 UTC
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Global effort launches to map the journey of rett syndrome
Knowledge-focused Recruiting nowThis study is creating a worldwide registry to collect information from caregivers about living with Rett syndrome. It aims to better understand the disease, track symptoms and treatments over time, and help researchers design better future studies. The data is collected remotely…
Matched conditions: RETT SYNDROME
Sponsor: Rett Syndrome Research Trust • Aim: Knowledge-focused
Last updated Mar 27, 2026 12:39 UTC
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Wearable tech trial aims to unlock better tracking for rare neurological disorder
Knowledge-focused Recruiting nowThis study is testing if special wearable sensors can reliably measure symptoms like heart rate, breathing, and sleep in people with Rett syndrome. About 30 participants will use the devices at home for several weeks and have one overnight clinic visit to compare results. The goa…
Matched conditions: RETT SYNDROME
Sponsor: Rett Syndrome Research Trust • Aim: Knowledge-focused
Last updated Mar 27, 2026 12:38 UTC