Updates

Teeth Misalignment Linked to Swallowing Difficulties in Girls with Rett Syndrome, Study Says
New Phase 2 Study on Anavex 2-73 for Rett Syndrome Begins in Australia
#AANAM – Experimental Gene Therapy AVXS-201 Shows Promise to Treat Rett Syndrome in Preclinical Studies
Overabundance of Nerve Fibers in the Skin May Affect Pain Sensitivity, Study Suggests
Study in Rats Provides Clues to Nerve Cell Connectivity and Body Movement Control
Mutations Causing Rett Syndrome Lead to Changes in Several Genes, Study Finds
‘Minds Wide Open’ Documentary Highlights Advances in Brain Research
Early Protein Alterations May Drive the Development of Rett Biomarkers
Physical Therapy and Other Interventions May Prevent Motor Deterioration in Patients with Rett Syndrome, Case Report Finds
Phase 3 Trial for Trofinetide Planned After Positive Phase 2 Results in Rett Syndrome
Better Preclinical Practices Would Boost Success in Clinical Setting for Rett Syndrome Therapies, Review Says
Parents of Girls with Rett Syndrome Need More Information About Treatment, Study Says
Annual Zoledronic Acid Injection May Improve Bone Health in Children with Rett Syndrome
Phase 2 Trial Evaluating Anavex 2-73 for Rett Syndrome Doses First Patient
Mosaic Mutations May Hold Key to Understanding Rett’s Development
Study Reports Rising Trend Of Prenatal Genetic Tests For Rett Syndrome
Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn
Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist
NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research
Understanding of How Females Inherit Traits May Hold Key to Treatment of X-linked Disorders
Misregulation in NPCs’ Development Might be Implicated in Rett Syndrome, Study Says
WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April
RettSyndrome.org Webinar Describes 2019 Research Priorities
StemoniX Develops 3D Human Microbrains of Rett Syndrome to Accelerate Therapy Development
Vienna to Host RARE2019 Meeting on Rare Diseases
Impaired Visual Search Affects Selective Attention in Rett Children, Study Shows
Basic Needs Take Up Large Chunks of Time in Daily Life of Teens and Young Adults with Rett Sydrome, Study Finds
Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide
Enrollment Complete for STARS Study of Sarizotan for Apnea in Rett Syndrome Patients
Rett Syndrome Research Trust Awards $10 Million to Researchers
Music Therapy Program Benefits Rett Patients, Parents, Study Shows
FDA Revising ‘Draft Guidance’ on Developing Treatments for Rare Diseases
Dentists Should be Aware of Oral Symptoms Related to Rett Syndrome, Study Recommends
Experiences with Rett Syndrome Described as ‘Obstacle Course’ by Caregivers, Study Shows
Girls with Rett Syndrome Display Atypical Brain Activity When Listening to Spoken Words, Study Finds
Rett Syndrome May Predispose Patients to High-fat Diets and Obesity, Study in Mice Finds
Adaptations Proposed to Help Assess Eye Tracking in Rett Syndrome Patients, Study Reports
Botulinum Toxin Reduces Excessive Drooling, Improves Oral Motor Function in Rett Syndrome Patients, Study Says
Anavex Proceeding with Phase 2 Trial to Assess Anavex 2-73 for Rett Syndrome

May 21, 2019May 21, 2019
Teeth Misalignment Linked to Swallowing Difficulties in Girls with Rett Syndrome, Study Says

Teeth malocclusion, or misalignment, is linked to severe dysphagia (difficulty in swallowing) in girls with Rett syndrome, a study finds. The research was reported in ... Read more
May 14, 2019May 14, 2019
New Phase 2 Study on Anavex 2-73 for Rett Syndrome Begins in Australia

A new Phase 2 clinical study to evaluate the investigational therapy candidate Anavex 2-73 for the treatment of Rett syndrome has started in Australia, ... Read more
May 10, 2019May 10, 2019
#AANAM – Experimental Gene Therapy AVXS-201 Shows Promise to Treat Rett Syndrome in Preclinical Studies

Preclinical data from mouse studies support initiating first-in-human clinical trials to explore the safety and effectiveness of the investigational gene therapy AVXS-201 in patients ... Read more
May 2, 2019May 2, 2019
Overabundance of Nerve Fibers in the Skin May Affect Pain Sensitivity, Study Suggests

Some people with Rett syndrome have an overabundance of nerve fibers associated with the senses in their skin, which may be linked to why some ... Read more
April 30, 2019April 30, 2019
Study in Rats Provides Clues to Nerve Cell Connectivity and Body Movement Control

A study in rats has provided new clues as to how the brain works to control body movements, which may have implications for disorders ... Read more

Mutations Causing Rett Syndrome Lead to Changes in Several Genes, Study Finds
April 23, 2019April 23, 2019
Mutations in the MECP2 gene, the underlying cause of Rett syndrome, affect genes that regulate several cellular ...
‘Minds Wide Open’ Documentary Highlights Advances in Brain Research
April 18, 2019April 18, 2019
In the documentary “Minds Wide Open,” which spotlights advances in brain research, Monica Coenraad is ...
Early Protein Alterations May Drive the Development of Rett Biomarkers
April 16, 2019April 16, 2019
Understanding protein alterations during the early phases of neuronal development may open the door ...
Physical Therapy and Other Interventions May Prevent Motor Deterioration in Patients with Rett Syndrome, Case Report Finds
April 11, 2019April 11, 2019
Physical therapy and other medical interventions, together with daily support from family and caregivers, ...
Phase 3 Trial for Trofinetide Planned After Positive Phase 2 Results in Rett Syndrome
April 9, 2019April 9, 2019
Investigational therapy trofinetide showed promising safety and efficacy results as a treatment for children and ...
Better Preclinical Practices Would Boost Success in Clinical Setting for Rett Syndrome Therapies, Review Says
April 4, 2019April 4, 2019
Discovery and development of therapies for Rett syndrome requires better preclinical research practices and study ...

Subscribe to our mailing list

Get regular updates delivered to your email box.

Thanks! Please check your email for a confirmation email.

Parents of Girls with Rett Syndrome Need More Information About Treatment, Study Says
April 2, 2019April 2, 2019
Clinicians should provide more support and information to parents of girls with Rett syndrome ...
Annual Zoledronic Acid Injection May Improve Bone Health in Children with Rett Syndrome
March 27, 2019March 27, 2019
One injection per year of zoledronic acid (sold under the brand name Reclast) can ...
Phase 2 Trial Evaluating Anavex 2-73 for Rett Syndrome Doses First Patient
March 21, 2019March 21, 2019
Mosaic Mutations May Hold Key to Understanding Rett’s Development
March 19, 2019March 19, 2019
Study Reports Rising Trend Of Prenatal Genetic Tests For Rett Syndrome
March 14, 2019March 14, 2019
Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn
March 14, 2019

Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist
March 12, 2019March 11, 2019
With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn ...
NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research
March 7, 2019March 5, 2019
Understanding of How Females Inherit Traits May Hold Key to Treatment of X-linked Disorders
March 5, 2019March 5, 2019
Misregulation in NPCs’ Development Might be Implicated in Rett Syndrome, Study Says
February 28, 2019February 28, 2019

WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April
February 26, 2019February 25, 2019
The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government ...
RettSyndrome.org Webinar Describes 2019 Research Priorities
February 26, 2019February 26, 2019
StemoniX Develops 3D Human Microbrains of Rett Syndrome to Accelerate Therapy Development
February 21, 2019February 21, 2019

Vienna to Host RARE2019 Meeting on Rare Diseases
February 21, 2019February 20, 2019
About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city ...
Impaired Visual Search Affects Selective Attention in Rett Children, Study Shows
February 19, 2019February 19, 2019
The first evidence that selective attention — the ability to focus on or select ...
Basic Needs Take Up Large Chunks of Time in Daily Life of Teens and Young Adults with Rett Sydrome, Study Finds
February 14, 2019February 14, 2019
Meeting basic daily tasks, from getting dressed to assuring good hygiene and medical care, are ...
Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide
February 12, 2019February 11, 2019
Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities ...
Enrollment Complete for STARS Study of Sarizotan for Apnea in Rett Syndrome Patients
February 7, 2019February 7, 2019
Patient enrollment is now complete for the pivotal STARS study investigating the safety, tolerability, ...
Rett Syndrome Research Trust Awards $10 Million to Researchers
February 5, 2019February 5, 2019
The Rett Syndrome Research Trust (RSRT) has awarded $10 million to a host of ...
Music Therapy Program Benefits Rett Patients, Parents, Study Shows
January 31, 2019January 31, 2019
A 6-month music therapy program improved social interaction and communication skills and reduced seizure ...
FDA Revising ‘Draft Guidance’ on Developing Treatments for Rare Diseases
January 30, 2019January 30, 2019
The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug ...
Dentists Should be Aware of Oral Symptoms Related to Rett Syndrome, Study Recommends
January 29, 2019January 29, 2019
Dentists should be aware that children diagnosed with Rett syndrome can develop several oral ...

Subscribe to our mailing list

Pin It on Pinterest