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…
A new clinical trial is starting for Friedreich's ataxia (FA). This trial will investigate if supplementation of vitamin D in its active form, also known as calcitriol, will aid in…
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…
Recent results of a Turkish trial have demonstrated the need for further research into IFN-gamma as a treatment for Friedreich's ataxia (FA). This study showed that the therapy resulted in…
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…
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…
According to an announcement from the Muscular Dystrophy Association, the organization has recently awarded a total of $1,076,232 in MDA Venture Philanthropy (MVP) funding towards the drug company AavantiBio, which…
The 2019 Global Genes Rare Patient Advocacy Summit was filled with incredible presentations that highlighted steps for progress in research and advocacy organization, provided solidarity for the rare community, and…
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…
The Beginnings Anne Pariser worked for 16 years at the FDA. For part of that time, she was a team leader for the Center for Drug Evaluation and Research where…
The Word "Rare" Unfortunately, many experts in rare disease believe the phrase "rare disease" may be harming the very population it describes. The Director of the National Center for Advancing…
According to a story from BioSpace, the biotechnology company Minoryx Therapeutics recently announced that the company has received official approval from the Spanish Agency of Medicines and Medical Devices (AEMPS)…
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For a long time there have been no treatments for Friedreich's ataxia. With no FDA approved therapies, people with this disease have only been receiving symptomatic treatment. Exicure has changed…
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.…
According to a story from globenewswire.com, the corporation Retrotope recently announced the initiation of single-patient, expanded access trials of its experimental product RT001. The patients involved have familial encephalopathy with neuroserpin…
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