Vivienne (“Vivi”) Horton has always been a bright and kind, albeit a bit shy and quiet, little girl. So her parents, Valerie and Terry, noticed when she wasn’t acting quite…
Stephanie Felix knew exactly what she wanted for her ninth birthday: to ring the bill at Beverly Knight Olson Children’s Hospital and signify that she was—officially!—cancer-free. When Stephanie was seven…
It’s incredibly important to raise rare disease awareness, spread education, and contribute to a more inclusive and equitable world. One of the best ways to start? Through children. Teaching the…
Although the medical field has made significant strides over the years, there is still a lot we don’t know about the body. For example, what can we learn about…
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…
From February 28 to March 2, rare disease advocates met in Washington, DC for the 12th Annual Rare Disease Week on Capitol Hill. Organized by Rare Disease Legislative Advocates (RDLA),…
Hand weakness, impaired circulation, pain and tightness in the neck, arms, and shoulders: while Jared Walsh, first baseman for the Los Angeles Angels, first started experiencing symptoms related to thoracic…
It’s no secret that diet and nutrition contribute greatly to our health. But how can dietary or nutritional changes help people who are living with certain illnesses? In the past,…
If current estimates are correct, around 10% of people with sarcoidosis within the United States are using off-label TNF-alpha inhibitors such as Remicade (infliximab). But according to an article in…
When Jen and Will Fox remember their son Dalton, they think about his infectious smile, his passion for adventures, and his absolute adoration of baseball. Dalton, who was diagnosed with…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
One of the symptoms of scleroderma is the formation of painful ulcers on the fingers. These ulcers are often difficult to manage; they do not respond well to treatment and…
Read Parts 1 and 2 of Katie's story, where we discuss the diagnostic journey, precocious puberty, and Katie's ZMYM2-related disorder diagnosis. Potential ZMYM2 Symptoms and the Need for Research The geneticist mentioned that symptoms…
At first, 52-year-old Lisa Wolff thought that she was dealing with sciatic pain. The pain was sometimes dull, sometimes burning, but generally manageable. When the pain became too much to…
February marked National Cancer Prevention Month. This month is designed to better understand, and raise awareness of, preventable causes of cancer; cancer warning signs; and different ways that we can…
In the 2009 draft, the Washington Nationals chose pitcher Stephen Strasburg as their first round pick. Over the years, Strasburg has started 247 times and holds a 113-62 record. This…
February 28th is recognized as Rare Disease Day around the world each year. This day is intended to bring recognition and awareness to the plight of the millions of people…
In March of 2021, twenty-eight-year-old Alix Burnard of Newbury, Berkshire UK had several Covid-19 tests due to a suspected infection. All three tests were negative, so she felt it…
Before you continue reading, make sure to check out Part 1 of Katie's story. True Precocious Puberty: The First Official Diagnosis The endocrinologist first ran a blood test, later followed with a…
In the United States, Orphan Drug designation is granted to drugs or biologics designed to treat, prevent, or diagnose rare conditions. “Rare” conditions refer to those affecting under 200,000…
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