Posts by: Forest Ray PhD

Forest Ray PhD

Forest Ray received his PhD in systems biology from Columbia University, where he developed tools to match drug side effects to other diseases. He has since worked as a journalist and science writer, covering topics from rare diseases to the intersection between environmental science and social justice. He currently lives in Long Beach, California.

February 1, 2021February 2, 2021

Newly Found RNA Molecules May Be Biomarkers for Disease Progression

News

A set of small molecules called microRNA (miRNA), found in a recent study, show potential both as biomarkers and as therapeutic targets of Gaucher disease. Determining differences in the levels ... Read more

January 26, 2021January 26, 2021

COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say

News, syndicated

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to ... Read more

November 2, 2020November 2, 2020

Potential Gene Therapy for Type 2 Gaucher, PR001, Placed on FDA Fast Track

News

The U.S. Food and Drug Administration (FDA) has given fast track designation to PR001, Prevail Therapeutics‘ investigational gene therapy for neuronopathic Gaucher disease. The FDA previously granted the therapy — now in an ... Read more

October 26, 2020October 26, 2020

Neuronopathic Gaucher Immune Response Might Protect Brain From Infection

News

Neuronopathic Gaucher disease (nGD) induces an immune response that protects against viral infections in the brain, according to an early study in animals. Understanding how this happens and whether GD ... Read more

September 3, 2020September 3, 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 “A ... Read more

August 25, 2020August 25, 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 input ... Read more

July 10, 2020July 10, 2020

National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US

News, syndicated

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in ... Read more

June 23, 2020July 28, 2020

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

News, syndicated

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will support ... Read more

June 10, 2020June 10, 2020

Coalition Will Address Racial Disparities in Rare Disease Communities

News

The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis for ... Read more

February 13, 2020February 13, 2020

FDA Grants Orphan Drug, Rare Pediatric Disease Designations to PR001 Investigational Gene Therapy

News

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Prevail Therapeutics‘ investigational therapy PR001 for the treatment of Gaucher disease. In addition, the regulatory agency granted ... Read more