Antisense Oligonucleotide Rescue of CGG Expansion–Dependent FMR1 Mis-Splicing in Fragile X Syndrome Restores FMRP
One of the most exciting advancements being done in Fragile XS research today is antisense oligonucleotide (ASO) therapy.
RECONNECT Trial Announces New AT-HOME Option for Participation
RECONNECT Trial Announces New AT-HOME/VIRTUAL Option for Participation
Project WellCAST: Caregivers of Children with Fragile X Needed for a Research Study
The Kelleher Lab at Purdue University is conducting a virtual, NIH-funded clinical trial called Project WellCAST. Project WellCAST aims to understand how to best support caregivers of children with rare neurogenetic conditions, including Fragile X syndrome.
Summary of the Fragile X Syndrome Basic Science Research session at the NFXF’s 18th International Fragile X Conference
Dr. Anubhuti Goel summarizes the Fragile X Syndrome Basic Science Research session at the NFXF's 18th International Fragile X Conference.
Summary of the Fragile X Premutation Panel Discussion at the 18th International Fragile X Conference
Dr. Connor Maltby, a 2022 Jr. Investigator, summarizes the Fragile X Premutation panel discussion at the NFXF's 18th International Fragile X Conference.
Summary of the Fragile X Messenger Ribonucleoprotein (FMRP) Series at the 18th NFXF International Conference
Dr. Talia Thompson summarizes the Fragile X Messenger Ribonucleoprotein (FMRP) series from the 18th NFXF International Conference
Summary of the Diagnosis and Treatment of Fragile X-Associated Tremor Ataxia Syndrome Panel Session
Dr. Michelle Tosin, a 2022 NFXF Junior Investigator, summarizes the Diagnosis and Treatment of Fragile X-associated Tremor Ataxia Syndrome session at the 18th International Fragile X Conference.
Summary of the Research Landscape Panel at the 18th International Fragile X Conference
Dr. Kate Shelly, a 2022 Jr. Investigator, summarizes the Research Landscape panel at the NFXF's 18th International Fragile X Conference.
Summary of the Fragile X Premutation Basic Science Research Session at the NFXF 18th International Fragile X Conference
Dr. Carolyn Yrigollen summarizes the Fragile X Premutation Basic Science Research session at the NFXF's 18th International Fragile X Conference.
Tetra Therapeutics: An Interview with Dr. Liz Berry-Kravis
Learn more about the ongoing Tetra trials in this 2-minute video with Dr. Liz Berry-Kravis
Antisense Oligonucleotide (ASO) therapy being explored in Fragile X syndrome!
A publication was released this week summarizing one lab’s discovery that could lead to a future treatment for Fragile X- ASO therapy.
Study: Language, executive function, and quality of life of those with the Fragile X premutation
Researchers at the University of Arizona are recruiting adult men living with the Fragile X premutation for a study on language, executive function, and quality of life.
Views and Experiences of Caregiver Coaching in Early Childhood Speech-Language Services: A Survey of Caregivers and Speech-Language Pathologists
The Research in Developmental Disabilities and Language Lab at The University of Wisconsin-Madison is conducting a survey to learn about you and your child’s experiences in speech therapy during early childhood.
Study: Behavioural and Emotional Outcomes in individuals with Neurodevelopmental Disorders (BEOND)
The Cerebra Network for Neurodevelopmental Disorders is conducting a survey to learn more about behaviour, social functioning, sleep, hyperactivity, mood, physical and mental health, as well as family functioning and wellbeing, and how these change over time.
Allos Pharma Inc. Receives FDA Guidance and Support for their Phase 3 Trial of Arbaclofen in Fragile X Syndrome
Allos Pharma Inc. received invaluable FDA feedback on the design of their Phase 3 trial for arbaclofen for the treatment of Fragile X syndrome.