Time

Fragile X: Unraveling Autism's Secrets

Cari Wheeler, center, and her dad Gary Boyer, seated, did not know they carried the fragile X trait until Max, center, was born with fragile X syndrome.Mikey Tnasuttimonkol for TIME They called him ...
Northcountrypublicradio.org

Fragile X held him back. An experimental drug is helping him break free

For 22 years, Jason Mazzola's life was defined by a genetic condition that can cause autism and intellectual disability. Then he started taking an... Fragile X held him back. An experimental drug is ...
Hosted on MSN

Fragile X Syndrome: Symptoms, Diagnosis, Treatments and Complications

Fragile X Syndrome: Symptoms, Diagnosis, Treatments and Complications: By Shreoshree Chakrabarty Fragile X Syndrome (FXS), also known as FX syndrome or fragile syndrome, is the most common inherited ...
Medical Xpress

Improperly processed genetic material in Fragile X syndrome—correctly cutting RNA offers a potential treatment

Fragile X syndrome is a genetic disorder caused by a mutation in a gene that lies at the tip of the X chromosome. It is linked to autism spectrum disorders. People with fragile X experience a range of ...

Fragile X deficits in mice respond to gene therapy

A gene therapy designed to replace a missing brain protein restored normal brain activity and improved behavior in a mouse ...
Medicine Buffalo

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

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 ...
Medical Xpress

Antisense therapy restores fragile X protein production in human cells

An antisense therapy developed by Joel D. Richter, Ph.D., Sneha Shah, Ph.D., and Jonathan K. Watts, Ph.D., at UMass Chan Medical School and Elizabeth Berry-Kravis, MD ...
Business Wire

Mirum Pharmaceuticals Announces First Patient Enrolled in the BLOOM Phase 2 Clinical Study Evaluating MRM-3379 in Fragile X Syndrome

FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today announced that the first participant has been enrolled in the BLOOM Phase 2 ...
NPR

An Alzheimer's Drug May Boost Cognition In People With Fragile X Syndrome

An experimental drug intended for Alzheimer's patients seems to improve both language and learning in adults with Fragile X syndrome. That is enough to change the lives of many people with Fragile X, ...
USA Today

Decades-long quest to beat Fragile X fueled by persistence, science and relentless optimism

NEWBURY, Mass. – Andy Tranfaglia seemed to be getting better by the day. He stopped vomiting his meals. His coordination improved enough that he could return a volley on the tennis court. His ...
The Conversation

Fragile X syndrome often results from improperly processed genetic material – correctly cutting RNA offers a potential treatment

For many people with fragile X, the mutated gene that causes symptoms is active rather than silenced. Thom Leach/Science Photo Library Fragile X syndrome is a genetic disorder caused by a mutation in ...