Neuren Pharmaceuticals will conduct a Phase 3 clinical trial for trofinetide, showing promise in treating Rett syndrome symptoms in children and adults. The trial aims to provide a treatment that addresses the underlying biology of the disease.
Rettsyndrome.org has funded six new research projects worth $775,000 to accelerate treatments and therapies for Rett syndrome. The projects focus on drug development and brain mechanisms underlying the disorder.
A Phase 2 clinical trial of trofinetide in girls with Rett syndrome aged 5-15 showed statistically significant clinical benefits, including improvements in the Rett Syndrome Behavior Questionnaire and Clinical Global Impression of Improvement. The high dose of trofinetide was well-tolerated with no dose-limiting effects observed.
Rettsyndrome.org has reached a milestone of $40 million in research funding for Rett syndrome treatments. The organization has funded over 40M in high-quality research grants and programs to date.
Rettsyndrome.org designates 14 US clinics as Clinical Research Centers of Excellence, fostering comprehensive care and partnering with families and healthcare providers. These centers accelerate research and treatment, providing critical support for individuals with Rett syndrome.
The NIH NCATS RDCRN funded 11 sites to study the natural history of Rett syndrome, MECP2 Duplication, CDKL5 disorder, and FOXG1 syndrome. Rettsyndrome.org announces an additional investment of $65,000 to add three more sites, bringing the total to 14, expanding geographical access and clinical infrastructure.
Rettsyndrome.org has made a financial commitment to support Phase 2 and Phase 3 clinical trials of trofinetide in adult women and children with Rett syndrome. The organization is hopeful that these trials will be successful and lead to the first potential treatments for Rett syndrome.
The Massachusetts Rett Syndrome Association and Rettsyndrome.org are partnering to support a Phase 2b clinical trial of IGF-1 for the treatment of Rett syndrome. The trial, funded by a $439,000 grant from Rettsyndrome.org, aims to develop new biomarkers and treatments for the condition.
Rettsyndrome.org announces new Translational Research Awards to tackle nonsense mutations in MECP2 gene, aiming to develop treatments for Rett syndrome. The awards include the Read-Through Program and Neuro-Habilitation grants, furthering research towards new pharmacologic treatments and improved clinical practices.
Rettsyndrome.org has awarded over $3.1 million in grants to researchers worldwide, focusing on basic, translational, and clinical research for Rett syndrome treatment and therapy development. The organization aims to accelerate research from discovery to medicine, covering a spectrum of Rett syndrome research.
The NIH has awarded a $29 million cooperative agreement to investigate four rare disorders: Rett syndrome, MECP2 Duplications, CDKL5 disorder, and FOXG1 syndrome. The study aims to understand the core clinical features of each disorder and identify factors that can modify their severity.
Rettsyndrome.org has awarded $1.5 million to support translational research and launch of the neuro-habilitation therapeutic program, focusing on cognitive therapies, physical therapies, and speech therapies to aid in skill development in Rett syndrome. The grants also fund clinical research to identify effective pharmacologic treatments.
The Rett Syndrome Association of Massachusetts has donated $150,000 to support a Rettsyndrome.org approved grant to Dr. Michela Fagiolini's project at the Boston Children's Hospital. The goal is to assess NMDA receptor modulators for potential treatments in girls diagnosed with Rett syndrome.
A Phase 1 clinical trial of mecasermin (rh-IGF-1) in girls with Rett syndrome demonstrated the treatment's safety and efficacy in ameliorating certain symptoms. The study suggests that IGF-1 could be a potential treatment for Rett Syndrome, paving the way for further research and possible FDA approval.
Key researchers propose standards and guidelines for Rett syndrome research to improve animal study design and transparency. The goal is to shorten the time to effective treatments and accelerate progress towards a cure.
Researchers successfully recreated Rett syndrome in adult mice by 'switching off' a critical disease-causing gene, challenging the notion that early expression of the gene protects against the development of symptoms. The study suggests therapies for Rett syndrome may need to be continuously maintained throughout an individual's life.
A team of researchers has developed a human cell-based model of Rett syndrome, overcoming the main limitation of accessing live neurons from patients. The study provides evidence of functional rescue using human cells and opens up new avenues for drug development and high-throughput screening.
Researchers halt life-threatening breathing arrests in mouse models of Rett syndrome using a combination of drugs. The breakthrough offers new hope for treating this devastating disease, which affects 1 in 10,000 young girls.
The International Rett Syndrome Foundation has awarded $1.5 million in research grants to advance understanding of the disorder and develop treatments. These awards will support innovative studies on the genetic and epigenetic mechanisms underlying Rett syndrome.
The 11th Annual Rett syndrome Symposium saw a record turn-out of nearly 150 attendees, including leading scientists, researchers, families affected by the disease, and volunteers. The event featured sessions on treatment strategies and Autism Spectrum Disorders, with new collaborations and relationships forged among participants.
A new form of the MECP2 protein has been discovered to be associated with Rett syndrome, a devastating neurological disorder affecting approximately 3,000 girls in the US. This breakthrough finding promises to aid in the diagnosis and treatment of Rett syndrome and other developmental disabilities.