New Gene-Editing Strategy Could Help Development of Treatments for Rare Diseases

Instead of requiring personalized gene edits for each patient, the new approach could create a standardized method to use for ...
STAT

Curing rare childhood diseases will falter unless Congress steps up

When our son Peter was diagnosed with an ultra-rare form of muscular dystrophy at the age of 10, the first question we asked was: Is there a treatment? The answer was no. By the time our daughter ...
STAT

The rare pediatric disease voucher program creates new treatments. I have new data to prove it

The Food and Drug Administration’s rare pediatric disease priority review voucher program, which has been providing incentives for lifesaving innovations since 2012, is doomed to disappear unless ...
Psychology Today

The Psychological Toll of Living with a Rare Disease

Rare diseases reveal that our sense of identity rests on fragile biological infrastructure, much of it operating outside ...

Researchers to test gene therapy for rare diseases with a new FDA trial protocol

Earlier this year, researchers at Children's Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to ...
Napa Valley Register

A look at some key statistics on rare diseases in the U.S.

The global pharmaceutical industry is make huge investments in treatments for rare diseases, which once drew little interest from major drugmakers. A look at some key statistics on rare diseases: ...
Business Wire

New Study Shows Impact of Ambry Genetics’ Patient for Life™ Program on Rare Disease Diagnosis

Peer-reviewed publication examines the benefits of proactive exome reanalysis on diagnostic outcomes Ambry Genetics’ Patient for Life program is supported by a dedicated team of scientists who ...

Struggling with a rare disease, a South Paris woman fights for care amidst a doctor shortage

Maine faces a severe shortage of health care workers, with projected gaps in primary care doctors and many nearing retirement ...
Sinar Daily on MSN

Rare Kidney Disease: The silent health crisis we need to talk about

For millions living with rare diseases, the road to diagnosis and treatment is often filled with uncertainty. It is also ...
Newsday

NYS needs rare disease advisory council

This guest essay reflects the views of Scott D. Reich, of Port Washington, whose nonprofit is developing a treatment for FOXG1 syndrome while providing support to the broader rare disease community.