After years of setbacks and uncertainty, Stealth BioTherapeutics has achieved a historic breakthrough with FDA approval of Forzinity (elamipretide), the first treatment for Barth syndrome, an ultrarare, life-limiting genetic disorder.…
Each year, Patient Worthy covers the American College of Medical Genetics conference, the leading source of both genetic education for health care professionals and a leading source of new information…
After years of determined advocacy and scientific perseverance, Stealth BioTherapeutics has achieved a landmark victory with the FDA’s accelerated approval of elamipretide (now branded as Forzinity) the first-ever treatment for…
Families and researchers in North Carolina are making urgent appeals to the FDA, highlighting the desperate need for faster approval of drugs for rare and ultra-rare diseases that threaten children’s…
The U.S. Food and Drug Administration (FDA) is reviewing the first medication specifically developed for Barth syndrome, a rare and life-threatening genetic disorder. As reported by STAT, this medication could…
After more than a decade of development, families say time is running out for children with Barth syndrome as the experimental therapy elamipretide faces yet another regulatory delay. Reported by…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
This opinion piece was provided by Kevin Woodward of Phoenix, Maryland. Kevin is a member of the Barth Syndrome Foundation board of directors. The role of the U.S. Food and…
The World Orphan Drug Congress USA 2023 was held from May 23-25, 2023 at the Gaylord National Resort & Convention Center in National Harbor, MD. This conference is focused solely…
According to a recent news release from PR Newswire, the European Medicines Agency (EMA) has recently awarded elamipretide its Orphan Drug designation for the treatment of Barth syndrome. This treatment,…
According to an article submitted to StatNews by members of the Barth Syndrome Foundation, the FDA approved the drug Aduhelm in accordance with its accelerated approval program. Although the treatment…
Rare disease research is often lacking due to a lack of funding, interest, or awareness. To combat this, the Barth Syndrome Foundation (BSF) and American Heart Association (AHA) have joined…
One-year-old Alexander was diagnosed with Barth syndrome before he was born; his parents knew to test for the condition after their son Elias passed away from complications in 2018. While…
THE 9TH INTERNATIONAL SCIENTIFIC, MEDICAL & FAMILY CONFERENCE "Power Up!" This event will be a convergence of health providers, researchers, and industry leadership to present the latest research and treatments…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
