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September 8, 2020September 23, 2020

Newly Discovered Mechanisms Controlling Memory, Learning Could Lead to Therapies

News

Scientists have discovered part of the mechanisms underlying synaptic plasticity — the process that allows nerve cell connections called synapses to become stronger or weaker over time — that ... Read more

September 1, 2020September 1, 2020

Same But Different Photo Contest Celebrates People With Rare Diseases

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Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme ... Read more

August 28, 2020August 28, 2020

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

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Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient ... Read more

August 25, 2020August 25, 2020

Better Communication Needed to Help Fragile X Patients Transition Into Adulthood, Study Suggests

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Better communication among different institutions is needed to assist young patients with fragile X syndrome make the transition from school into adult life, according to a small study. Support ... Read more

August 21, 2020August 21, 2020

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

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The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. ... Read more

August 18, 2020August 18, 2020

Fewer ‘Sensing Antennas’ in Brain Nerve Cells May Contribute to Fragile X, Mouse Study Asserts

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Having fewer primary cilia — cellular structures that act like antennas to detect several signals — in nerve cells may contribute to learning and memory deficits in fragile X syndrome, ... Read more

August 14, 2020August 14, 2020

New Streaming Channel Showcases Rare Disease Films

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When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the ... Read more

August 4, 2020August 4, 2020

NIH Grant Supports Research Into Potential Targets for Fragile X Therapies

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Investigators at the Mercer University’s College of Pharmacy were awarded almost $425,000 in three-year funding to advance work into serotonin receptors as potential targets for preventing seizures in people ... Read more

July 24, 2020July 24, 2020

Zygel Improves Caregiver Impressions of Behavioral Symptoms in Some Fragile X Children

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The experimental cannabidiol (CBD) gel Zygel eased behavioral problems in children with fragile X syndrome and benefited those with greater disease severity according to their caregivers, new clinical trial data show. The ... Read more

July 21, 2020July 21, 2020

Blood Proteins May Help Measure Effectiveness of Metformin in Fragile X

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Measuring the levels of three blood biomarkers could help identify patients with fragile X syndrome who are likely to benefit from treatment with the diabetes medicine metformin, a study suggests. The ... Read more

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