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 February 2024, Jacob Bell reported in Biopharma Dive that pharmaceutical company Takeda Pharmaceuticals is planning on rapidly advancing late-stage testing of TAK-861 for people with type 1 narcolepsy (also…
In 2020, Chris and Susan Finazzo received news that would change the trajectory of their lives. They learned that their sons Chase (now 9) and Dylan (now 6) had a…
In early March 2024, Clinical Trials Arena reports that global biopharmaceutical company Bristol Myers Squibb shared new data from the Phase 3 DAYBREAK study. Within the study, researchers explored how…
In the rare disease community, fundraising plays a pivotal role in driving forward research and treatment initiatives, offering vital support to affected families, and guaranteeing access to necessary resources and…
Corticosteroids are one of the standards-of-care for treating alopecia areata, alongside treatments such as topical sensitizers and Rogaine. However, none of these treatments have a high level of evidence in…
Children with late-infantile onset Batten disease (CLN2) may be treated with an enzyme replacement therapy (ERT). However, this therapy cannot reverse the damage or progression children have already experienced. Researchers…
At the end of January 2024, global healthcare leader Eli Lilly and Company shared that its therapy OLUMIANT (baricitinib) received regulatory approval in Canada for the treatment of severe alopecia…
The 20th annual WORLDSymposium took place from February 4-9, 2024. During the course of the research conference, at least one presentation centered on data from the Phase 2 Mini-COMET long-term…
Having a chronic illness can be isolating. It can be difficult to share vulnerable and sensitive information about yourself with others, or to find ways to manage the trauma and…
The 17th Annual Congress of the European Association for Haemophilia and Allied Disorders took place this year from February 6-9, 2024. During the Congress, stakeholders came together to discuss how…
When Jason Fox was just a child, doctors told his family that he would most likely not live to 18 years old. He had been diagnosed with Duchenne muscular dystrophy…
Imagine if you had recurring attacks of severe and unexplained swelling beneath your skin. For individuals living with hereditary angioedema (HAE), a rare inherited disorder, this isn't just a mere…
Having a rare disease can be challenging and isolating. It's often difficult to describe the experience to someone who hasn't gone through it. Mobilizing funds for support, resources, and research…
Alzheimer’s disease is a progressive neurodegenerative disease and the most common form of dementia. People with Alzheimer’s disease may have difficulty remembering information, problems with learning, sleep disruptions, poor judgment,…
Zachary Thomas has been an advocate for the mucopolysaccharidosis type I (MPS I) community since he was born. Newborn screening is a public health initiative that tests newborn babies for…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
In total, there are thirteen subtypes of Batten disease. Batten disease, also known as neuronal ceroid lipofuscinosis (NCL), is a rare and inherited neurodegenerative disorder that causes visual impairment, behavioral…
It was never just about reaching the summit of Aconcagua, the highest mountain in both the Western and Southern hemispheres. For Scott Osleeb, scaling the heights of this remarkable peak…
What role do brain alterations play in the onset and progression of spinocerebellar ataxia type 6 (SCA6)? While previous studies highlighted cerebellar changes in individuals with SCA6, the underlying mechanisms…
Developing novel therapeutics for rare diseases is increasingly important. Understanding the unmet needs of rare disease communities and providing effective, accessible therapies can significantly improve the lives of those affected.…
A majority of rare diseases are genetic in nature, with an estimated 50-75% of these conditions manifesting in childhood. The Rare Pediatric Disease designation was established to encourage and streamline…
When it comes to rare disease, rare isn't actually that rare. More than 10,000 rare diseases have been identified. Rare diseases affect more than 350 million people across the globe…
In late January 2024, the U.S. Food and Drug Administration approved a label expansion for Dupixent (dupilumab). Dupixent, a monoclonal antibody targeting IL-4 and IL-13, is currently used to treat…
Quinnlyn Walters is a bright, bubbly, and active 8-year-old. She loves reading, riding horses, swimming and softball, karate, Star Wars, and spending time outside with her friends and family. But…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
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