Advancements in Pediatric Treatment Phase 3 Results of ...

Agios Announces Results from Phase 3 ACTIVATE-KidsT Study of ...

With our focus on lifelong, debilitating rare diseases, we hope that this study will be the first of several pediatric studies to make a ...

Press Release: Dupixent positive phase 3 data in children one to 11 ...

Paris and Tarrytown, NY, June 26, 2024. The New England Journal of Medicine has published results from a positive phase 3 study of Dupixent ( ...

Phase 3 Open-Label Clinical Trial of Elexacaftor/Tezacaftor/Ivacaftor ...

Taken together, these results demonstrate that ELX/TEZ/IVA treatment improves CFTR function in children aged 2–5 years. These results also suggest that earlier ...

Pfizer Announces Positive Topline Results From Phase 3 Study of ...

(NYSE: PFE) today announced positive topline results from the Phase 3 AFFINE study (NCT04370054 ) evaluating giroctocogene fitelparvovec, an ...

Phase 3 Trial of Crinecerfont in Pediatric Congenital Adrenal ...

In this phase 3 trial, crinecerfont was superior to placebo in reducing elevated androstenedione levels in pediatric participants with CAH.

Dupixent® (dupilumab) Positive Phase 3 Data in Children 1 to 11 ...

(NASDAQ: REGN) and Sanofi today announced the New England Journal of Medicine (NEJM) has published results from a positive Phase 3 trial for ...

Design of a phase 3, randomized, double-blind, placebo-controlled ...

The efficacy endpoints used in this study will differ from those used in adults and in the previous pediatric phase 3 trial; they have been adapted to be more ...

Results From Phase 3 LAVENDER Trial Announced

The drug has also received a rare pediatric disease designation from the agency. A decision is expected from the FDA by March 12, 2023, on what could be the ...

Vertex Advances Inaxaplin (VX-147) into Phase 3 Portion of ...

“Advancing this trial into Phase 3 and broadening the trial to include younger patients is a critical step forward in bringing this potential ...

Press Release: NEJM publishes ALTUVIIIO XTEND-Kids phase 3 ...

NEJM publishes ALTUVIIIO XTEND-Kids phase 3 data supporting its potential to transform the treatment landscape for children with severe ...

Pfizer Provides Update on Phase 3 Study of Investigational Gene ...

About the Fordadistrogene Movaparvovec Clinical Program · About Duchenne Muscular Dystrophy · About Pfizer: Breakthroughs That Change Patients' ...

Dupixent® (dupilumab) Phase 3 Trial Shows Positive Results in ...

(NASDAQ: REGN) and Sanofi today announced that a Phase 3 trial assessing the investigational use of Dupixent® (dupilumab) in children aged 1 to ...

Phase 3 Trial of Epicutaneous Immunotherapy in Toddlers with ...

The primary efficacy end point result was observed in 67.0% of children in the intervention group as compared with 33.5% of those in the placebo ...

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 ...

Gene therapy in pediatrics – Clinical studies and approved drugs ...

The results of ongoing clinical trials (NCT02702115 , NCT03041324 ) shows evidence of genome editing and favorable safety profile in patients ...

2-year results from an open-label, phase 3 extension study - Nature

This phase 3 extension study was conducted to document the efficacy and safety of continuous, daily vosoritide treatment in children with ...

What's new in pediatrics - UpToDate

Outline · ORTHOPEDICS AND SPORTS MEDICINE · Long-term outcomes of Little League elbow (September 2024) · GENERAL PEDIATRICS AND ADOLESCENT MEDICINE · ALLERGY, ...

Merck and Moderna Initiate Phase 3 Study Evaluating V940 (mRNA ...

KEYTRUDA is indicated for the adjuvant treatment of adult and pediatric (12 years and older) patients with stage IIB, IIC, or III melanoma ...

Advancements in FDA approvals for pediatric obesity treatment

GLP-1 receptor agonists, used to treat diabetes, are now also being used for obesity in children and adolescents. ... Pediatric obesity affects ...

Recent progress in the treatment of cancer in children - Butler - 2021

Therefore, attention has shifted to the use of molecularly targeted agents and immunotherapies to improve the outcomes of children who are not ...