Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.
Before you read on, make sure to check out Part 1 of Kristin and Kayden's story. In Part 1, Kristin discusses the diagnostic journey and how Kayden was diagnosed with a dermal…
Continue ReadingTethered Spinal Cord, Dermal Sinus Tract, and Chiari Malformation: How Kayden’s Health Journey Inspired his Mother to Take Action (Pt. 2)
Charlie and Megan Hieb would do anything to help their daughters, Nora and Lucy. They would truly go to the ends of the earth to ensure that their daughters…
Previously known as Marburg hemorrhagic fever, Marburg virus disease (MVD) is a rare but serious viral hemorrhagic fever with sporadic outbreaks occurring in South Africa, Ghana, Zimbabwe, Uganda, Kenya,…
Continue ReadingEquatorial Guinea Faces First Marburg Virus Disease (MVD) Outbreak
The Lashoff family loves living in St. Louis; they’ve been there for six years and can’t get enough of it, from watching hockey at the Enterprise Center to marveling at…
Continue ReadingTethered Spinal Cord, Dermal Sinus Tract, and Chiari Malformation: How Kayden’s Health Journey Inspired his Mother to Take Action (Pt. 1)
A rare disease diagnosis can conjure up a multitude of emotions, from relief at finally learning what is going on to fear or isolation when trying to figure out what…
Continue ReadingFighting for Recognition: Why Kate Continues to Advocate for PTEN Hamartoma Tumor Syndrome Awareness
Before you read on, don't forget to check out Part 1 of Jennifer's story. In Part 1, Jennifer shared her diagnostic journey and the four year stretch it took her to receive…
Pancreatic cancer unfortunately comes with a poor prognosis. It is the third leading cause of cancer-related deaths within the United States, though it is on track to become the second…
Continue ReadingFive-Year Survival Rate Rises for Pancreatic Cancer
When Pamelia J. first realized that her breasts were growing in size, she was kind of excited about it. Pamelia already had large breasts (a J-cup) but really felt…
Continue Reading“My Breasts Grew 4 Sizes in 8 Months”: A Story of Gigantomastia
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…
Continue ReadingEA-2353 for Retinitis Pigmentosa (RP) Granted Fast Track Designation
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…
Continue ReadingLearning to Live with Spontaneous Periodic Hypothermia: Lachlan’s Story (Pt. 2)
A case of systemic sclerosis. Source: Nevit Dilmen / CC BY-SA (https://creativecommons.org/licenses/by-sa/3.0)
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.…
Continue ReadingBelimumab Granted Orphan Drug Designation for Systemic Sclerosis
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…
Continue Reading“We May Bend, But Never Break”: How the Chandler Family Faces a Tuberous Sclerosis Complex (TSC) Diagnosis with Resilience
Photo courtesy of Mark, Donna, and Lachlan Lasikiewicz
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,…
Continue ReadingLearning to Live with Spontaneous Periodic Hypothermia: Lachlan’s Story (Pt. 1)
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,…
Continue ReadingWriting the Book: How Nicole’s Journey Continues to Raise ALPS Awareness (Pt. 2)
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…
Continue ReadingAdvocate with Multiple Myeloma Works to Raise Awareness of Racial Disparities in Cancer Care
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…
Continue ReadingKetarx for Rett Syndrome Earns Orphan Drug Designation
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…
Continue ReadingWriting the Book: How Nicole’s Journey Continues to Raise ALPS Awareness (Pt. 1)
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;…
Continue ReadingAn Expandable Prosthesis Has Helped a Boy with Osteosarcoma Gain Freedom
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…
Continue ReadingBEA-17 Earns Orphan Drug Designation for Glioblastoma
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…
Continue ReadingEfzofitimod Granted Orphan Drug Designation for Sarcoidosis in the European Union
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…
Continue ReadingBachelor Zach Shallcross Shares that He Was Born with Pyloric Stenosis
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…
Continue ReadingTAKHZYRO Now FDA-Approved for Children Aged 2-12 with HAE
The 2023 Special Olympics BC Winter Games took place from February 2-4, 2023. Sporting events at Winter Games include 5-pin bowling, curling, figure skating, alpine or cross-country skiing, and more!…
Continue ReadingTeen with Noonan Syndrome Competes at 2023 Special Olympics BC Winter Games
Known as “Slydah,” Chicago White Sox relief pitcher (RP) Liam Hendriks has done amazing things on the field. In September/October 2021, Hendriks was named the American League Reliever of…
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