On June 21, 2023, the Rare Disease Legislative Advocates (RDLA) hosted its monthly webinar. These webinars help provide updates to the rare disease community on legislation and other policy initiatives…
As she stood in the garden, surrounded by friends and family, a splitting headache richocheted through Ciara Wilkie's head. Her vision blurred. At first, Ciara didn't think much of it.…
In 2015, rare disease stakeholders in North Carolina established the first Rare Disease Advisory Council (RDAC); the National Organization for Rare Disorders (NORD) explains that an RDAC is an…
In the average person, you would expect to see normal to near-optimal low-density lipoprotein (LDL) cholesterol levels sitting at 129 mg/DL or lower; levels under 100 are seen as healthier.…
Treatment options such as Haegarda and Takhzyro have significantly improved the treatment landscape for people living with hereditary angioedema (HAE). The treatments, used as prophylaxis, work to reduce disease burden…
Raising awareness for rare diseases and conditions is important for many reasons. First, it helps to increase education and understanding around these rare conditions. Awareness also offers support to those…
Caleb Castle-Parker was born ten weeks premature. At the time, his parents didn’t know how his prematurity might affect him (if at all) long-term. But Caleb seemed to be…
Aboard the private yacht, the crew and passengers delighted in the sights of the open water. It was relaxing onboard: a welcomed break from the pressures of daily life. The…
In August 2022, Payton Quaife’s doctor recommended running some blood tests. The doctor didn’t want to frighten the family. But Quaife had a number of bruises that were healing poorly…
uniQure, a leading gene therapy company, recently shared an update on a Phase 1/2 clinical study evaluating AMT-130 for Huntington’s disease. The safety and proof-of-concept study enrolled 26 participants with…
Uplifting Athletes, a national nonprofit organization that harnesses the power of sport to support people impacted by rare diseases, celebrated 20 years of Lift for Life with Penn State University…
When it comes to rare diseases, advancing research is crucial to developing a better understanding of the disease and how it can be treated. However, it can sometimes be difficult…
For as long as he can remember, Eli McCausland has been fascinated by wrestling. His passion for the sport eventually culminated in his co-ownership of Midwest All-Star Wrestling, as well…
When it comes to the rare disease landscape, there is not enough conversation discussing mental health needs. People undoubtedly need more mental health support and resources during the diagnostic odyssey.…
In August 2022, I first interviewed Ryan Sheedy, the Co-Founder of mejo, a company dedicated to creating tools and resources designed to support caregivers and parents. Sheedy discussed how…
In 2019, South Carolina Governor Henry McMaster signed Dylan’s Law into effect. This law was passed in the wake of Dylan Emery’s death. The young boy had Krabbe disease, a…
Have you ever had an itch that you just can't seem to scratch? Intensely itchy skin, also known as pruritus, is a hallmark characteristic of many conditions that affect the…
Did you know that June is considered Dravet Syndrome Awareness Month? Every year, people within the Dravet syndrome community come together to amplify stories, raise awareness, and encourage the general…
At the very top of the Rensselaer Polytechnic Institute's website reads a bold statement and call to action: "Why not change the world?" As one of the United States' most…
The World Orphan Drug Congress USA 2023 was held from May 23-25, 2023 at the Gaylord National Resort & Convention Center in National Harbor, MD. This conference is focused solely…
In late May 2023, clinical-stage biopharmaceutical company Sangamo Therapeutics, Inc. (“Sangamo”) shared that its product isaralgagene civaparvovc (ST-920) was granted Fast Track Designation by the FDA. This designation is…
A positive opinion is needed from the Committee for Medicinal Products for Human Use (CHMP), part of the European Medicines Agency (EMA), to begin marketing a drug. Once a positive…
WebMD recently carried a story about a young woman who, after nine years of wheelchair confinement caused by a rare metabolic disease with no name, was able to walk…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
As clinicians and scientists continue to delve into RNA sequencing benefits, they are learning how RNA sequencing provides insights for people with rare genetic conditions. The learning experience will provide…
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