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.
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
Clinical trials are an important tool within the healthcare realm. These trials assist in developing deeper understandings of diseases, assessing and discovering treatment options, and learning new ways of identification…
Continue ReadingTop-Line Data Available from Elamipretide Trial for Geographic Atrophy
The Food and Drug Administration (FDA) has four distinct approaches to expedite the availability of therapies for rare, severe, or life-threatening illnesses: Priority Review, Breakthrough Therapy, Fast Track, and Accelerated…
Continue ReadingLBS-008 for Stargardt Disease Receives Fast Track Designation
For the last 52 years, individuals with Wilson disease have been relying, largely, on the same treatment options. These options, designed to remove copper from the body, include Cuprimine (penicillamine).…
In Europe, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare, life-threatening, or chronically debilitating conditions. For the European Union (EU), a “rare”…
Continue ReadingTSHA-120 Earns Orphan Drug Designation in Europe for Giant Axonal Neuropathy
Currently, there is no way to determine when a relapse will occur in relapsing and remitting multiple sclerosis (MS), one of the two main forms of MS. However, this may…
Continue ReadingQuanterix Blood Test for MS Earns Breakthrough Devices Designation
Dr. Omar P. Haqqani, MD FACS is currently the Chief of Vascular and Endovascular Surgery at the Vascular Health Clinics. He has a keen interest in understanding and addressing vascular…
Continue ReadingDr. Haqqani Unpacks Why New Research Improves PAH Survival
It’s finally May – and you know what that means! That’s right, this month is Huntington’s disease (HD) Awareness Month. The theme for this month’s HD Awareness Month is #LetsTalkAboutHD.…
Continue ReadingMay 11: Kick of HD Awareness Month with a Live Storytelling Event!
Rare Pediatric Disease designation is granted by the FDA to drugs or biologics intended to treat life-threatening diseases affecting individuals aged 18 or younger. In this case, rare conditions are…
Continue ReadingEDIT-301 for Beta Thalassemia Earns Rare Pediatric Disease Designation
When Krista Webb was just two years old, doctors determined that she had some level of hearing loss. Her family was surprised about this; nobody else in her family, and…
Prednisone is one of the more common corticosteroids prescribed to individuals with lupus to help reduce inflammation and decrease symptom burden. Corticosteroids are also prescribed for a number of other…
Continue ReadingCorticosteroids May Increase SCD Hospitalization Risk
Don't forget to check out Part 1 of our interview with Katheron Intson, where we discussed her background, her research experience in GRIN1, and the reasons behind why she developed…
Continue ReadingAccelerating the Journey to Rare Disease Treatment: An Interview with Katheron Intson of Varient (Pt. II)
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