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.
When Sophia Scott was growing up, her parents noticed that she was displaying a variety of different potentially concerning traits. They began undergoing some tests. Eventually, just one day before…
Continue ReadingFamily Hopes to Raise Sanfilippo Syndrome Awareness through #ChaseTheSigns Campaign
The Retina World Congress, a premier congress to discuss issues in retinal health, recently took place from May 12-15, 2022. During the Congress, various speakers and stakeholders discussed advances…
Continue ReadingRetina World Congress: Pars Plana Victrectomy Could Help Control Uveitis
What happens when you treat your condition, but the medication isn’t effective? Sometimes, other approved medications are beneficial for patient use, but other times, there are no approved medications.…
Continue ReadingJyseleca for Ulcerative Colitis Approved in Scotland for NHS Coverage
In Europe, Orphan Drug designation is granted to therapies intended to treat, prevent, or diagnose a rare, life-threatening, or debilitating disease. For the purpose of this status, rare conditions…
Continue ReadingDevimistat for Biliary Tract Cancer Earns EMA Orphan Drug Designation
The journey to a rare disease diagnosis can be long and daunting - and, in some cases, may mean facing a few misdiagnoses first. According to KTVB7, this is what…
Continue ReadingA Family Raises Tubb4a Leukodystrophy Awareness: Killian’s Story
When they found out that they were having a son, Nash and Callie Horne were thrilled. They even decided to name their son Nash as well. But then an unexpected…
Continue ReadingA Mom is Undergoing In-Utero Surgery to Fix Her Baby’s Spina Bifida
Fast Track designation is designed to facilitate the development of, and expedite the review of, drugs intended to treat serious conditions and fill unmet patient needs. This designation comes alongside…
Continue ReadingOfranergene Obadenovec for Ovarian Cancer Earns Fast Track Designation
Her brother, her sister, and her father - all people that Salima Mohip lost before they turned 43. Each of them passed away from heart attacks, a consequence of their…
Continue ReadingWoman with Familial Hypercholesterolemia Treated with Apheresis
It’s no secret that teachers can make a huge impact on the students in their classrooms. And that’s just what Judy Griffin did for her student Ashley. When Judy started…
Continue ReadingTeen with Huntington’s Disease Gifted New Wheelchair
In the past, researchers have observed a relationship between hemophilia and bone-related issues. However, while this observation is helpful, there have been few studies examining the prevalence of bone problems…
Continue ReadingHigh Rate of Bone Problems in Female Hemophilia Carriers
In the United States, the FDA grants Breakthrough Therapy designation to drugs intended to treat serious and life-threatening illnesses, and/or drugs which show significant improvement over available therapies. After receiving…
Continue ReadingICYMI: EDSIVO for vEDS Earns Breakthrough Therapy Designation
Jörg Richter is no stranger to difficult bike rides. In fact, shares KRDO, Richter has ridden his bike across the United States - from coast to coast - seven times…
Continue ReadingCoast-to-Coast Bike Ride to Raise Money for Care-for-Rare
When David and Melissa Wickham found out that they were expecting, the pair was thrilled. They had already been together for years – it took seven to conceive their daughter,…
Continue ReadingHelp the Wickham Family to Support their Daughter with an Omphalocele
We’ve reached the month of May, and you know what that means – it is Guillain-Barre syndrome (GBS) and Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) Awareness Month! During this month, the…
How does our body fight infections? After we get infected with a serious illness, in what ways does our immune system prepare? Understanding these processes could help hone treatments and…
Perry Cox is feisty, full of energy, and loves taking part in new activities with her friends. According to her father Griffin, Perry, now five years old, loves dancing and…
Continue Reading“High Hopes” Aims to Help Others with Pediatric Cancer
Adolescence is the phase which exists between childhood and adulthood; therefore, people who are between ages 10-19 are typically considered to be adolescents. How does adolescence change the way people…
Continue ReadingAdolescents with JIA Have Longer Rheumatology Wait Times
In 1996, the World Federation of Hemophilia (WFH) launched its Humanitarian Aid Program; it later expanded the program in 2016 to better reach and support patients with hemophilia. The program…
Continue ReadingCSL Behring to Donate Coagulation Factor Therapy to WFH Humanitarian Aid Program
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions. A rare disease is one affecting under…
Continue ReadingValbenazine for HD Chorea Granted Orphan Drug Designation
Have you ever heard of West Nile virus? In many cases, this mosquito-borne illness does not cause severe issues. However, in just about 1% of cases, West Nile virus can…
During the Phase 1/2 SUNRISE clinical trial, which evaluated LB-001 for pediatric patients with methylmalonic acidemia, at least two participants experienced serious and severe adverse reactions: thrombotic microangiopathy. While investigating…
Continue ReadingFDA Lifts Clinical Hold on LB-001 IND for Methylmalonic Acidemia
According to Fox News, the Connecticut Department of Public Health recently shared that there was at least one diagnosed case of Powassan virus within the state. Typically, it is rare…
Continue ReadingConnecticut Reports First Powassan Virus Case This Year
From 2010-2013, Abi Phillips embraced the role of Liberty Savage on Hollyoaks, a British fictionalized TV series about the lives of teenagers and their families. The program has since been…
Continue Reading28-Year-Old Hollyoaks Star Diagnosed with Thyroid Cancer
On the first Sunday in May, Cincinnati, OH hosts the Flying Pig Marathon – its 26.2 mile race. This year marked the 24th annual event. But for 17-year-old Diego Ramirez,…
Continue ReadingBoy with DMD Finishes his 11th Flying Pig Marathon
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