News

September 18, 2020September 18, 2020

Hospitalization After Emergency Department Visit Common in Fragile X Patients, Study Finds

News

Almost half of emergency department visits for fragile X syndrome result in hospitalizations, according to a nationwide study in the U.S. The finding underscores the importance of surveillance systems ... Read more

September 15, 2020September 15, 2020

Zynerba, FDA to Discuss Next Steps for Zygel in Fragile X Treatment

News

The U.S. Food and Drug Administration (FDA) and Zynerba Pharmaceuticals will meet later this year to discuss the results of the CONNECT-FX trial and next steps regarding a potential ... Read more

September 11, 2020September 11, 2020

Closing Leaky Mitochondria Halts Behavioral Problems in Fragile X, Study Suggests

News

Shortage of energy due to leaky mitochondria — the powerhouses of cells — contributes to behavioral abnormalities in fragile X syndrome, a study has found. Closure of the leaky ... Read more

September 8, 2020September 8, 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 — which ... Read more

September 1, 2020September 1, 2020

Same But Different Photo Contest Celebrates People With Rare Diseases

News, syndicated

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

News, syndicated

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

News

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

News, syndicated

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

News

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

News, syndicated

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

Pin It on Pinterest