As reported in Business Wire, Vico Therapeutics has initiated patient dosing in an expanded cohort of its Phase 1/2a clinical study evaluating VO659, an antisense oligonucleotide (ASO) designed to target…
Editor's Note: Patient Worthy is honored to share this post from Martha Harlam, Director of the Solution Project. Are you a student or researcher passionate about the future of neurology?…
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…
Written by Martha Harlam My journey with Ataxia has been a life full of ups and downs. Ataxia has defined most of my life since it was first diagnosed in…
The future of healthcare is going to be changed and impacted by the use of artificial intelligence (AI), hopefully in beneficial ways. According to an article from McMaster University, researchers…
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…
September 25th is International Ataxia Awareness Day! The National Ataxia Foundation (NAF) has great resources to help spread the word and show our solidarity for the Ataxia patient community; on…
The 62nd NAF Annual Ataxia Conference "Ataxia: A Treatable Disease" Are you affected by ataxia? Then don't miss out on the largest ataxia themed event in the world where patients…
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…
According to a new study, people who suffer from rare disease are more prone to suffer from levels of anxiety, depression, pain and fatigue. The findings are proof that special…
This article was originally published on Luke's blog and is shared here with permission. Keeping our kids safe is every parent’s number one job. Even as an adult I associate…
"Success largely depends on two factors. Number one is each individual person's decision to take action. Number two is our interaction with each other and support of each other in…
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