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…
A University of Florida research team has announced its results of a gene replacement therapy in the treatment of Barth syndrome, which delivered promising results in its initial phase of testing.…
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…
Barth Syndrome What is Barth syndrome? Barth syndrome is a rare, metabolic, and neuromuscular disorder that occurs exclusively in males, since it is passed from mother to son through the X chromosome. The…
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…
As 2025 winds down, the pharmaceutical industry is bracing for a series of pivotal FDA decisions that could redefine treatment paradigms and reshape competitive dynamics across several major therapeutic areas.…
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…
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…
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