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'You think that can't be my kid, that can't be what is wrong with them...' Young Wabash Valley girl battles rare disease

'You think that can't be my kid, that can't be what is wrong with them...' Young Wabash Valley girl battles rare disease
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New Drug Discovered to Treat Intellectual Disabilities

New Drug Discovered to Treat Intellectual Disabilities
by Pooja Shete on
December 17, 2020 at 12:58 PM
Fragile X Syndrome is a genetic disorder which cause mild to moderate intellectual disability. It is one of the leading causes of autism. A new drug has been discovered which is the first drug for the treatment for Fragile X Syndrome.
The research was conducted as collaboration between University at Buffalo and Tetra Therapeutics.
The drug BPN14770 showed positive results in phase 2 clinical study and improved cognitive function in adult male patients with Fragile X Syndrome.
According to the Centers for Disease Control and Prevention,
Fragile X Syndrome is the most common cause of inherited intellectual disability and there is no cure for it. ....

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New drug moves closer to becoming first treatment for Fragile X Syndrome

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BUFFALO, N.Y. - A new drug discovered through a research collaboration between the University at Buffalo and Tetra Therapeutics took a major step toward becoming a first-in-class treatment for Fragile X Syndrome, a leading genetic cause of autism.
The drug, BPN14770, achieved positive topline results in a phase 2 clinical study. The innovative treatment improved cognitive function in adult male patients with Fragile X Syndrome.
Fragile X Syndrome - a genetic disorder for which there is no cure - is the most commonly known cause of inherited intellectual disability, according to the Centers for Disease Control and Prevention.
We are very excited about the results of this study, said Mark Gurney, PhD, founder and chief executive officer of Tetra Therapeutics. In addition to being safe and well tolerated, treatment with BPN14770 led to significant cognitive improvement, specifically in the language domains, and we also saw a clinically meaningful benefit i ....

Elizabeth Berry Kravis , Mark Gurney , Jamesm Odonnell , Ying Xu , Centers For Disease , Rush University Medical Center , Ub School Of Pharmacy , Research Foundation , University At Buffalo , Tetra Therapeutics , Fragilex Syndrome , Disease Control , Pharmaceutical Sciences , எலிசபெத் பெர்ரி கிராவிஸ் , குறி கர்னி , யிங் ஐயூ , மையங்கள் க்கு நோய் , அவசரம் பல்கலைக்கழகம் மருத்துவ மையம் , ுப் பள்ளி ஆஃப் மருந்தகம் , ஆராய்ச்சி அடித்தளம் , பல்கலைக்கழகம் இல் எருமை , டெட்ரா சிகிச்சை , நோய் கட்டுப்பாடு , மருந்து அறிவியல் ,