This paper discusses the role fragile X mental retardation protein (FMRP) plays in protein expression in astrocytes, and suggests that FMRP loss in astrocytes may contribute to the development of fragile X.
Institute for Basic Research/ The Seaver Autism Center
New York Fragile X Clinical Trials Accepting Participants
(The following are four separate trials):
Trials at IBR (Institute for Basic Research) on Staten Island
IBR on Staten Island is now screening adolescents as well as adults for a trial of AFQ056 from Novartis. Males aged 12-45 who have Fragile X may be eligible. This trial runs for 4 months and offers an extension phase in which participants can take the drug free of charge.
IBR is also running a trial of arbaclofen from Seaside Therapeutics for boys and girls ages 5-11 who have Fragile X; this trial includes an extension phase as well. Seaside has completed enrollment for their trials of arbaclofen for ages 12-50.
Researchers link Fragile X syndrome protein to 93 genes that have been implicated in ASD. Lead investigator says the findings may lead to more detailed genetic tests.
Researchers led by Dr. Ben Philpot, an ASF funded mentor, at UNC School of Medicine found that seizures in individuals with Angelman syndrome could be linked to an imbalance in brain cell activity. Angelman syndrome exhibits frequent comorbidity with autism spectrum disorders.
Researchers are conducting advanced trials of the first drugs expressly designed to correct the genetically induced signaling problems in the brain that result in autism. The early indications are positive enough to offer new hope for families and spark interest from drug companies.
Researchers are conducting advanced trials of the first drugs expressly designed to correct the genetically induced signaling problems in the brain that result in autism. The early indications are positive enough to offer new hope for families and spark interest from drug companies.
