Neuromyelitis optica spectrum disorder (NMOSD) can have long-term impacts for those affected. Current research suggests that up to 69% of patients may have severe unilateral or bilateral vision loss…
Enzyme replacement therapy (ERT) has been explored as a potential therapeutic option for mucopolysaccharidosis type III A (MPS III-A or Sanfilippo syndrome type A). However, these solutions have not…
What would you do if you were told that your child had a rare disease? How about if your child had an ultra-rare disease, which affects fewer than 20 in…
The other day I read a post in a dystonia support group that is similar to something I see and hear very often. I’m sure you have as well, and…
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…
Over the past few decades, research has shown a potential connection between metabolic disorders and multiple sclerosis (MS). While this connection has been made, anti-hyperglycemic medications (often used to…
When Leonidas Boyne was born, he seemed—for all intents and purposes—to be a healthy, happy child. At five months old, he is still a bubbly, happy baby with a smile…
Before you read on, make sure to check out Part 1 of Emily's story. In Part 1, Emily discusses what Stickler syndrome is and her diagnostic journey. Today, we'll discuss coming to…
There are limited therapeutic options for people living with autosomal dominant polycystic kidney disease (ADPKD); currently, the only real standards-of-care include dialysis and kidney transplantation. When discussing the therapeutic landscape,…
If you’ve ever wanted to run around Philadelphia in your underwear for a good cause, I have an event to tell you about! As first reported by the Philly…
The American Society of Hematology (ASH) held its 64th Annual Meeting in December 2022, during which time a variety of medical stakeholders discussed issues, trends, and research in the…
In the reality television world, Megan Marx is best known for her time on Bachelor Australia, Bachelor in Paradise, and The Challenge Australia. But Marx is about to be…
Little 2 1/2-year-old Raiden Pham cannot walk or talk, but he can smile at his parents and radiate love. The baby’s parents are on a 24/7 schedule caring for…
Living with a rare genetic disease like Stickler syndrome (Or “Sticklers”) isn’t a death sentence. If Emily Katharine wants people to know one thing, that’s it: that they are more…
In a late December 2022 news release from biopharmaceutical company Altimmune, Inc., the company shared that positive topline data was available from a 24-week study—including a 12-week extension—which evaluated…
Jenny Decker’s disease, Charcot-Marie-Tooth (CMT), is named after the three doctors who first discovered the disease in 1886. CMT is a progressive neurological disease that affects approximately 2.6…
Jason Tolson age 30 of Yorkshire, UK had not been diagnosed when he first began to have symptoms in May 2022. Jason began to experience muscle and joint pain…
Throughout his professional career, Marcus Stewart played in over 500 soccer games, scored more than 250 goals, and played at clubs including Ipswich Town, Sunderland, Bristol City, Exeter City, and…
Right now, there are only an estimated 10 people in Ireland who have been diagnosed with a form of mucopolysaccharidosis type I (MPS I) called Hurler syndrome. This is the…
Nipah virus was first discovered in 1999 following an outbreak in people and pigs in Malayasia in Singapore. Since then, the zoonotic disease has spread, causing nearly annual outbreaks in…
In the past, Katie Lockwood has worked in a variety of different roles within counseling and education; she even worked as a disability advocate, championing inclusion on a larger…
The coronavirus pandemic has significantly altered not just the world we live in, but the way we engage within this new world. It has been difficult and isolating to…
High-risk neuroblastoma can be difficult to treat in children. Some research suggests that between 40-50% of children with high-risk neuroblastoma relapse following treatment, causing poor outcomes. When Flora Gentleman was…
When Charlotte Harris was born, she seemed - for all intents and purposes - to be a healthy infant. But her mother Melissa became concerned about a potential health issue…
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…
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