According to a recent article by The Manila Times, Design Therapeutics has reported encouraging interim results from its ongoing Phase 1/2 RESTORE-FA study, highlighting both clinical improvement and biomarker activity…
PTC Therapeutics announced that the US Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) regarding the company’s New Drug Application (NDA) for vatiquinone, an investigational therapy intended…
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…
It was never just about reaching the summit of Aconcagua, the highest mountain in both the Western and Southern hemispheres. For Scott Osleeb, scaling the heights of this remarkable peak…
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…
According to a story from GlobeNewswire, the gene therapy company LEXEO Therapeutics has recently announced that its investigational gene therapy LX2006 has been granted both Orphan Drug designation and Rare…
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…
A recent announcement in Biospace by Capsida Biotherapeutics and CRISPR Therapeutics of their newly-formed partnership offers renewed hope for amyotrophic lateral sclerosis (ALS) and Friedreich’s Ataxia patients. The companies believe…
Sometimes, medical studies just don't go the way that companies want them to. According to MedCity News, this is what biotechnology company Larimar Therapeutics ("Larimar") was faced with during…
In a press release from late February 2021, biopharmaceutical company Retrotope shared that its lead drug candidate, RT001, received Rare Pediatric Disease designation for the treatment of patients with Friedreich's…
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…
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…
According to a press release from Barcelona-based biotechnology company Minoryx Therapeutics, the Company has completed enrollment for a phase 2 clinical study of its experimental Friedreich's ataxia treatment, MIN-102 (generic…
According to a story from Ysios Capital, the biotechnology company Minoryx Therapeutics recently announced that the first patient has been dosed in the company's phase 2 clinical trial. This trial…
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.…
Many times, diseases can take very physical tolls on a person’s body. These physical symptoms can sometimes be uncomfortable, they can be painful, and sometimes they may even be embarrassing…
According to a story from Rare Revolution Magazine, Georgia Hart was first diagnosed with Friedrich's ataxia when she was 15 years old. Since then, living with this rare disease has…
According to a story from Charcot-Marie-Tooth Disease News, the U.S. Food and Drug Administration (FDA), under the leadership of commissioner Scott Gottlieb, has begun to change clinical trials by accepting…
My name is Alan. I cross many borders as a person with a rare disease. These borders are physical, geographical and psychological. Crossing so many borders has earned me the…
© 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.
