Fragile X treatment KER-0193 gets FDA orphan, rare disease tags
The FDA has granted orphan drug and rare pediatric disease designations to fragile X treatment KER-0193, Kaerus Biosciences said.| Fragile X News Today
The FDA has granted orphan drug and rare pediatric disease designations to fragile X treatment KER-0193, Kaerus Biosciences said.| Fragile X News Today
Long-term treatment with ZYN002 cannabidiol gel eased symptoms of irritability in children and adolescents with fragile X syndrome, per trial data.| Fragile X News Today
Kaerus is planning a proof-of-concept Phase 2 trial for KER-0193 after its fragile X syndrome treatment was found safe in healthy adults.| Fragile X News Today
Zygel safely led to behavioral improvements among children with fragile X syndrome, including those likely to have severe disease.| Fragile X News Today
A U.S. study shows that repeated sampling of hair follicles in people with fragile X syndrome can be used to measure FMRP protein levels.| Fragile X News Today
Due to the variability of its symptoms, a conclusive diagnosis of fragile X syndrome can only be obtained with genetic testing.| Fragile X News Today
Tetra Therapeutics has amended the protocols of two ongoing clinical trials testing zatolmilast to improve access for fragile X patients.| Fragile X News Today
The FDA has approved a Phase 2 trial to test SPG601, an oral treatment candidate, in men with fragile X syndrome.| Fragile X News Today
The LivJoy Foundation is funding a four-year postdoctoral fellowship for a scientist at USC who will study fragile X syndrome in females.| Fragile X News Today
A combination of ibudilast and gaboxadol was shown to normalize behavior and improve memory in a mouse model of fragile X syndrome.| Fragile X News Today
Blood levels of two proteins, known as MMP9 and APP, could serve as biomarkers for fragile X syndrome where genetic testing is not possible.| Fragile X News Today
School age-girls with fragile X show significantly poorer cognitive function and academic performance relative to matched healthy girls.| Fragile X News Today
Sensory symptoms, including overreactions to stimuli, are very common among fragile X patients and affect their daily life, a study found.| Fragile X News Today
Learn about fragile X syndrome, the most common cause of inherited intellectual disability and autism spectrum disorders.| Fragile X News Today
