- Positive Top|line Results for Phase 3 Trofinetide Trial🔍
- Clinical Trial News & Updates🔍
- Neurogene Reports Positive Interim Efficacy Data from First Four ...🔍
- Acadia Pharmaceuticals Announces Positive Top|line Results from ...🔍
- Potential Rett syndrome drug shows promising results in phase 3 trial🔍
- Positive top|line results from pivotal Phase 3 trial in Rett syndrome🔍
- Trofinetide for the treatment of Rett syndrome🔍
- Taysha Gene Therapies Announces Positive Clinical Data Across ...🔍
Positive Top|Line Rett Syndrome Clinical Trial Results
Positive Top-line Results for Phase 3 Trofinetide Trial
Positive top-line results for trofinetide began years ago when IRSF recognized treatment potential in Rett syndrome and made major financial ...
The following companies are actively conducting clinical trials in individuals with Rett syndrome. This stage may take 6-7 years to complete the 3 phases of ...
Neurogene Reports Positive Interim Efficacy Data from First Four ...
All participants experienced a 2-point improvement in the clinician-rated Clinical Global Impression-Improvement (CGI-I) scale from baseline ...
Trofinetide has been granted Fast Track Status and Orphan Drug Designation for Rett syndrome. Trofinetide has also been granted Rare Pediatric ...
Potential Rett syndrome drug shows promising results in phase 3 trial
A potential first-of-its-kind drug to treat the symptoms of Rett syndrome received positive top-line results from a phase 3 trial.
Both programs have Fast Track designation from the US Food and Drug Administration (FDA). Neuren has granted an exclusive licence to Acadia ...
Trofinetide for the treatment of Rett syndrome: a randomized phase ...
In this phase 3 study in a large cohort of girls and women 5–20 years of age with RTT, trofinetide demonstrated a statistically significant ...
Taysha Gene Therapies Announces Positive Clinical Data Across ...
Additionally, the patient showed improvement in posture and stability at week 25 post-treatment. We believe these longer-term clinical data ...
Neurotech reports more positive Phase 1/2 Rett Syndrome clinical ...
On April 17, clinical-stage biotech Neurotech International (ASX:NTI) reported top-line results for the Phase 1/2 Rett Syndrome clinical trial ...
Neurogene's Rett Syndrome Gene Therapy NGN-401 Demonstrates ...
“Data were also concordant across multiple scales and show consistency of effect across patients, despite their unique clinical presentations at ...
Double-blind, randomized, placebo-controlled study of trofinetide in ...
To determine safety, tolerability, and pharmacokinetics of trofinetide and evaluate its efficacy in female children/adolescents with Rett syndrome (RTT), ...
Rett syndrome drug studied at Vanderbilt approved for patients
The first drug to treat the symptoms of Rett syndrome was recently approved, following a Nature Medicine study publishing results from the ...
Positive Top-Line Rett Syndrome Clinical Trial Results - Listcorp
About NTIRTT1 The NTIRTT1 Phase I/II clinical trial will examine the effects of daily oral treatment of NTI164 and is targeting the recruitment ...
Neurotech reports positive top-line Phase 1/2 Rett Syndrome clinical ...
Special Report: Neurotech International has reported positive top-line Phase 1/2 Rett Syndrome clinical trial results with its NTI164 being the ...
Trofinetide for the treatment of Rett syndrome: Long-term safety and ...
Trofinetide improved the symptoms of Rett syndrome with a manageable safety profile in LAVENDER, a 12-week, placebo-controlled, phase 3 study.
Anavex Life Sciences Provides an Update on Rett Syndrome Program
When looking at other placebo-controlled Rett syndrome trials, ANAVEX®2-73 compares favorably in terms of absolute RSBQ improvements, with the ...
Long-term Daybue led to continual improvements in patients: Studies
... Rett syndrome in extension studies, data show ... That approval was based mainly on positive data from a Phase 3 clinical trial ...
Rett Syndrome: The Emerging Landscape of Treatment Strategies
Trofinetide, the first drug approved by the FDA (March 2023) for treatment of individuals with RTT, was the result of a series of studies first ...
Rett Syndrome: Reaching for Clinical Trials - ScienceDirect
Rett syndrome (RTT) is a syndromic autism spectrum disorder caused by loss-of-function mutations in MECP2. The methyl CpG binding protein 2 binds ...
Rett Syndrome and the Role of MECP2: Signaling to Clinical Trials
There is large diversity in location; however, there are two gene-focused clinical trials, gene therapy and gene editing. Furthermore, trofinetide has shown ...