Fragile X

Minocycline Promising in Fragile X Syndrome

Source: 
Medscape Today
Date Published: 
September 7, 2010
Abstract: 

Parents of children with fragile X syndrome report that minocycline led to positive improvements in language, attention levels and behavior. They also report experiencing adverse side effects such as mild gastrointestinal issues and some increased irritability.

Cambridge's Seaside at Forefront of new approach to Fragile X

Source: 
The Boston Globe
Date Published: 
August 23, 2010
Abstract: 

The story of Matthew, a 9-year-old with Fragile X Syndrome, is one of the first patients on one of the first medications ever developed specifically to address the causes of an autism-like disorder. And — at least for him — it seems to be working.

Scientists Identify New Drug Strategy Against Fragile X Syndrome

Source: 
Science Daily
Date Published: 
August 10, 2010
Abstract: 

Researchers at Emory University School of Medicine have identified a potential new strategy for treating fragile X syndrome -- the most common inherited cause of intellectual disability. The researchers have found that a class of drugs called phosphoinositide-3 (PI3) kinase inhibitors can correct defects in the anatomy of neurons seen in a mouse model of fragile X syndrome.

UT Southwestern researchers uncover fragile X syndrome gene's role

Source: 
EurekAlert
Date Published: 
May 7, 2010
Abstract: 

Researchers at UT Southwestern Medical Center have discovered how the genetic mutation that causes Fragile X syndrome, the most common form of inherited mental retardation, interferes with the "pruning" of nerve connections in the brain. They found Fragile X is caused by a mutation in a single gene, Fmr1, on the X chromosome. The gene codes for a protein called FMRP, which plays a role in learning and memory but whose full function is unknown. The protein's role in pruning nerve connections had been unclear.

Seaside Therapeutics Secures $30 Million Financing

Source: 
Reuters
Date Published: 
September 17, 2009
Abstract: 

Seaside Therapeutics LLC today announced that it has secured $30 million in financing from a private, family investment firm which is committed to advancing research in the field of autism and Fragile X Syndrome.

Fragile X: Translation in Action

Source: 
Neuropshcyopharmacology, Bear, Dolen et al
Date Published: 
2008

Fragile X is a synapsopathy--a disorder of synaptic function and plasticity. Recent studies using mouse models of the disease suggest that the critical defect is altered regulation of synaptic protein synthesis. Various strategies to restore balanced synaptic protein synthesis have been remarkably successful in correcting widely varied mutant phenotypes in mice. Insights gained by the study of synaptic plasticity in animal models of fragile X have suggested novel therapeutic approaches, not only for human fragile X but also for autism and mental retardation of unknown etiology.