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.

September 29, 2020September 29, 2020

Givinostat Wins Rare Pediatric Disease Designation; Phase 3 DMD Trial Fully Enrolled

News

Italfarmaco has now completed recruitment — delayed in the spring due to the COVID-19 pandemic — of boys with Duchenne muscular dystrophy (DMD) for its ongoing Phase 3 trial of givinostat, ... Read more

September 3, 2020September 3, 2020

September is Duchenne Action Month

News

September is Duchenne Action Month, an awareness, fund-raising, and educational campaign supporting people with Duchenne muscular dystrophy (DMD) Hosted by Parent Project Muscular Dystrophy (PPMD), each week of the ... Read more

September 2, 2020September 2, 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 25, 2020August 25, 2020

Viltepso Commercially Available in the US for DMD Treatment

News

NS Pharma‘s Viltepso (viltolarsen) is now commercially available in the U.S. for people with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. The FDA conditionally approved Viltepso earlier this month, ... Read more

August 24, 2020August 24, 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 20, 2020August 20, 2020

First Patients Enroll in Phase 1 Trial of EPM-01, Potential Becker MD Therapy

News

A Phase 1 clinical trial of EPM-01 ((+)-epicatechin), a potential oral treatment of Becker muscular dystrophy that targets cellular energy, has enrolled its first patients. This one-year study of Epirium Bio‘s small ... Read more

July 28, 2020July 28, 2020

Stem Cell Therapy VTA-110 Named FDA Orphan Drug for DMD Treatment

News

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Vita Therapeutics‘ VTA-110, a candidate therapy for people with Duchenne muscular dystrophy (DMD). The designation grants ... Read more

July 23, 2020August 4, 2020

Benitec Begins Early Study of BB-301 Gene Therapy for OPMD

News

Benitec Biopharma has begun the first study to support the start of clinical trials of BB-301, a potential gene therapy for people with oculopharyngeal muscular dystrophy (OPMD). The BB-301 Tissue Transduction ... Read more

July 16, 2020July 16, 2020

NS Pharma Opens Support Hub to Help DMD Patients Access Treatments

News

NS Pharma has launched a support hub, called NS Support, for people in the U.S. with Duchenne muscular dystrophy (DMD) and their healthcare providers. NS Support will provide patients and ... 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 ... Read more