Jacklyn and Derrick Shaw have supported each other through some of the toughest and most complicated situations that anyone could go through, from a rare disease diagnosis to the loss…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
Six years ago, the United MSD Foundation began its annual Zebra Run with a goal of raising multiple sulfatase deficiency (MSD) awareness, as well as garnering funds to advance…
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…
According to a story from Globe Newswire, the gene therapy company uniQure recently announced that it has received conditional marketing authorization for its gene therapy etranacogene dezaparvovec (marketed as HEMGENIX®).…
Each year, March 1 marks the start of Myeloma Action Month; during this month, the International Myeloma Foundation (IMF) encourages people to get involved, share stories, raise awareness and…
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.
