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An Alzheimer s Drug May Boost Cognition In People With Fragile X Syndrome
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An Alzheimer s Drug May Boost Cognition In People With Fragile X Syndrome
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An experimental treatment produced improvements in cognitive function and language in patients with fragile X syndrome, according to study results published on April 29 in
Nature Medicine. Fragile X syndrome (known as FXS for short) is the most common known genetic cause of autism and the most common cause of inherited intellectual disability. These results offer hope for patients with fragile X syndrome and their families, said Elizabeth Berry-Kravis, MD, PhD, a pediatric neurologist at Rush University Medical Center and principal investigator of the study. The majority of clinical outcome measures were in favor of the drug. These measures included performance-based assessments, biomarkers, and parent and physician-rated scales, which in combination, suggest a meaningful impact on the global FXS disease process.
Alongside the race for Covid-19 treatments and vaccines, the industry has maintained momentum
2020’s headlines have been dominated by coronavirus since the first reports of a mysterious virus causing pneumonia in Wuhan, China, in January. The pharmaceuticals sector’s response was huge. While the pharma landscape has been somewhat unusual this year, at least some business has carried on.
The need for effective and innovative ways to treat disease is something that even a global pandemic will not blunt
Of course, a lot of the focus on clinical trials this year has been Covid-19-related, but trials continue elsewhere, despite the challenges and delays the pandemic has caused. However, there has been disruption. According to Evaluate Pharma’s World preview 2020, outlook to 2026 report, ‘Hundreds of clinical trials have been mothballed and trial readouts delayed; smaller developers in particular are exposed here, as they rely on fresh data for financial injections and partnerin
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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