In the United States, Fast Track designation is granted to drugs designed to treat rare and/or serious conditions and that fill an unmet medical need. The process aims to…
Jennifer Reid is no stranger to rare or chronic illnesses. Over her lifetime, she grappled with Guillain-Barre syndrome (GBS) in 2000; it took nearly thirteen years of treatment and rehabilitation…
Before you read on, make sure to check out Part 1 of our story. In Part 1, Lachlan's mom Donna discusses the long diagnostic journey, what symptoms Lachlan was showing, and how…
February 15th is recognized annually as International Angelman Day, a day dedicated around the world to spreading awareness among the general public and in the medical field about Angelman syndrome,…
Their son, Caeleb, was only eleven months old when he developed a severe bleeding episode. The family had just moved from Houston to New Mexico and they had not yet…
Kimberly Greenberg’s baby, Parker, was born a few weeks early but came into this world on January 2, 2016, as a healthy, normal baby. However, just before Parker was to…
Initially, belimumab (sold under the brand name BENLYSTA) was approved for the treatment of patients for lupus and lupus nephritis. It was the first FDA-approved biologic treatment within this indication.…
Gavin Chandler loves toy cars, watching Big City Greens, fans, the “Baby Shark” song, and anything that lights up or makes noise—including, his mother Jessica shares, the vacuum cleaner. He…
When Lachlan Lasikiewicz turned gray one day, his lips a frightening blue, his grandma and aunt didn’t panic. They simply wrapped him up in a blanket and held him close,…
Before you read, don't forget to check out Part 1 of our interview with Laura, Nicole's mom. In Part 1, we discuss Nicole's diagnostic journey, what acute lymphoproliferative syndrome (ALPS) is,…
For as long as she can remember, Tiffany Williams wanted to make a difference, especially in the healthcare field. She earned her doctorate degree and began working as a…
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…
In the United States, Orphan Drug designation is granted by the FDA to drugs focused on the treatment, prevention, or diagnosis of rare conditions; these are those affecting fewer than…
I recall the day, almost six years ago, when I observed the liveliness of joy in one who paraded around in a pink frilly skirt, smiling with merriment while wearing…
Four-year-old Rayhan Majid of North Lanarkshire, UK was a healthy little boy who enjoyed swimming, football, and Taekwondo. He began to have severe headaches in October 2017. His mum,…
Two and a half years — that is how long it took for the Roublick family to learn that their daughter Nicole had an extremely rare condition called autoimmune lymphoproliferative…
Ever since the first moment he can remember, eight-year-old Dominic Gamez was drawn to travel soccer. He felt strong and powerful as his legs carried him across the field;…
In the past, it was difficult to incentivize the development of treatments for rare diseases, given issues with both profitability and small patient populations. However, all people are deserving…
Whole genome sequencing is providing high-level information about new rare diseases putting pressure on drug developers to pick up the pace. Of approximately 7,000 rare diseases, most do not have…
Hello, I'm Ailbhe and along with Adam, my husband, and Sam (5) and Dara (3), we are the O'Reilly family from Dublin, Ireland. Dara has Glut1 deficiency syndrome and was…
Over 30 million people in the European Union (EU) are living with a rare disease. Yet rare disease drug development is still somewhat stifled. Orphan designation helps to incentivize drug…
The most dramatic. Season. EVER!!! If you've ever watched "The Bachelor" or "The Bachelorette," you've probably heard this refrain from the show's host, talking up the ridiculous antics that keep our eyes…
My name is Becky Tilley. I live in the UK, am married, and my wonderful husband Carl and I have three children: Isabella (age four), Joshua (age two), and Avary…
In the past, there have been limited therapeutic options for pediatric patients living with hereditary angioedema (HAE). Children aged 2 to <6 years old had no approved prophylaxis therapy. For…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…
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