August 2025 was a landmark month for rare disease innovation, as the U.S. Food and Drug Administration (FDA) granted four historic approvals, each representing a first for their respective conditions,…
From Diagnosis to Determination: My Friedreich Ataxia Journey I was always clumsy. I could never walk in a straight line down the street, bumping into my parents and getting told…
Three years after the U.S. Food and Drug Administration (FDA) first placed a full clinical hold on Larimar Therapeutics’ Phase 2 clinical study, the hold has officially been lifted. The…
In Gibson City, IL, students at Gibson City-Melvin-Sibley High School (GCMS High School) are fighting to find a cure for Friedreich’s ataxia (FA). According to reporting from Jared Thomas…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
Our genes dictate our health in many ways. When working properly, genes encode for the production of various proteins and enzymes throughout the body. So when genes become mutated,…
Until recently, the only therapeutic options for people with Friedreich ataxia were symptomatic and focused on symptom management: mobility aids, surgery, psychotherapy, speech therapy, heart medications, hearing and vision…
Dr. Valeria Ricotti of the Institute for Child Health- Great Ormond Street UK, spoke with BBC News on the subject of AI analyzing body movement. Dr. Ricotti and other researchers…
Christian Maugee and Shandra Trantham met as undergraduates at the University of South Florida (USF) and immediately clicked. Their first chance meeting was in the disability office. It was here…
Recently, hundreds of people celebrated the 12th Annual Fara 5k in Providence, Rhode Island. This race was organized over a decade ago in an effort to raise money for Friedreich…
The FDA grants designations, such as the Rare Pediatric Disease and Orphan Drug designations, to aid in the drug development process. Recently, LEXEO Therapeutics has received these two designations for…
GeneDx, an OPKO Health company, has recently launched several genetic tests. Within this group of tests are repeat expansion analysis for Friedreich ataxia, spinocerebellar ataxia (SCA), and other forms of…
In a press release from late January 2021, biopharmaceutical company Seelos Therapeutics, Inc. ("Seelos") announced the issuance of an Australian patent for Trehalose (SLS-005). The patent (#2019204513) centers around the…
An Australian royal commission is investigating claims that children with disabilities are being excluded from online learning, according to The Guardian. They are also looking into issues with the services…
Cystinosis Cystinosis is a rare disease which is caused by a buildup of cystine in the body's cells. This buildup impacts various organs including the brain, eyes, liver, kidneys, and muscles.…
According to Newswise, new findings indicate that electrical stimulation of the spinal cord and brain may be effective in treating symptoms of a group of rare neurological movement disorders, neurodegenerative ataxias.…
When you have a rare disease like Friedreich ataxia (FA), which affects only 50,000 people in the United States, it is pretty easy to feel alone and isolated. Usually, the…
Kiara Trussell thought her dreams of being a cheerleader would never happen. This is partly because Friedreich’s ataxia has made her wheelchair bound. Friedreich’s ataxia is a progressive neurological condition.…
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