Updates

March 21, 2019March 21, 2019
Phase 2 Trial Evaluating Anavex 2-73 for Rett Syndrome Doses First Patient

The first patient has been dosed in Anavex Life Sciences’ Phase 2 trial evaluating Anavex 2-73 for the treatment of Rett syndrome. The trial (NCT03758924) assessing the safety, tolerability, ... Read more
March 19, 2019March 19, 2019
Mosaic Mutations May Hold Key to Understanding Rett’s Development

Genetic screening for mosaic mutations — those present in only some cells in the body — may be useful to understand disease development and severity ... Read more
March 14, 2019March 14, 2019
Study Reports Rising Trend Of Prenatal Genetic Tests For Rett Syndrome

A new study reports an increasing trend in the number of prenatal genetic tests to detect spontaneous mutations that can cause conditions such as ... Read more
March 14, 2019
Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn

Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as ... Read more
March 12, 2019March 11, 2019
Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist

With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases ... Read more

NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research
March 7, 2019March 5, 2019
Rare diseases affect about 30 million Americans — roughly the same number as those ...
Understanding of How Females Inherit Traits May Hold Key to Treatment of X-linked Disorders
March 5, 2019March 5, 2019
Researchers have discovered the genetic mechanisms involved in female inheritance and X chromosome silencing. This process, ...
Misregulation in NPCs’ Development Might be Implicated in Rett Syndrome, Study Says
February 28, 2019February 28, 2019
Proteomic analysis — the characterization of all the proteins present in a cell at ...
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
Rettsyndrome.org is dedicated to funding research in every stage of development and every aspect ...
StemoniX Develops 3D Human Microbrains of Rett Syndrome to Accelerate Therapy Development
February 21, 2019February 21, 2019
Three-dimensional microbrains built in the lab using stem cells from Rett syndrome patients can ...

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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
Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide
February 12, 2019February 11, 2019
Enrollment Complete for STARS Study of Sarizotan for Apnea in Rett Syndrome Patients
February 7, 2019February 7, 2019
Rett Syndrome Research Trust Awards $10 Million to Researchers
February 5, 2019February 5, 2019

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
Dentists Should be Aware of Oral Symptoms Related to Rett Syndrome, Study Recommends
January 29, 2019January 29, 2019
Experiences with Rett Syndrome Described as ‘Obstacle Course’ by Caregivers, Study Shows
January 24, 2019January 24, 2019

Girls with Rett Syndrome Display Atypical Brain Activity When Listening to Spoken Words, Study Finds
January 22, 2019January 22, 2019
Like typically developing children, girls with Rett syndrome can distinguish known words from meaningless ...
Rett Syndrome May Predispose Patients to High-fat Diets and Obesity, Study in Mice Finds
January 17, 2019January 17, 2019
Adaptations Proposed to Help Assess Eye Tracking in Rett Syndrome Patients, Study Reports
January 15, 2019January 15, 2019

Botulinum Toxin Reduces Excessive Drooling, Improves Oral Motor Function in Rett Syndrome Patients, Study Says
January 10, 2019January 10, 2019
Botulinum toxin treatment for excessive salivation — known as hypersalivation or sialorrhea — in ...
Anavex Proceeding with Phase 2 Trial to Assess Anavex 2-73 for Rett Syndrome
January 8, 2019January 8, 2019
After the U.S. Food and Drug Administration recently approved an investigational new drug application ...

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