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.
Max Moore normally loved going on family walks with his mom Mandie, dad Devin, and brother Chase (age four at the time) throughout their neighborhood. But when he was eight…
In 2006, shortly after giving birth to her second child, Kelly Knight’s life drastically changed. Debilitating headaches and rapid weight gain, especially around her neck and shoulders, left her feeling…
When it comes to medical research, diversity, equity, and inclusion need to be larger parts of the conversation. As it stands now, medical research can be fiercely hierarchical, expensive, and homogenous in terms of…
Also known as the most common form of eczema, atopic dermatitis is a chronic pruritic skin condition that causes skin to become excessively itchy, dry, cracked, and discolored. Atopic dermatitis…
Having a rare disease, or a family member with a rare disease, can be expensive. Management often requires multiple visits to specialists. Medication and medical equipment are not always covered…
In Wickenburg, Arizona, the community sticks together. Community means strength. So when Noah Ahnlund was diagnosed with a rare autoimmune disease called membranoproliferative glomerulonephritis type II (MPGN II/dense deposit disease)…
May is Vasculitis Awareness Month. Vasculitis refers to a group of rare and often challenging diseases characterized by blood vessel inflammation. These conditions can affect anyone, at any age, and…
In the United States, Orphan Drug designation is granted by the FDA to drugs or biologics designed to treat, diagnose, or prevent a rare condition affecting fewer than 200,000 people…
Acknowledgment: This patient story is sponsored by Gilead Oncology and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing relevant,…
Do you know what your macula does? The macula, or the central portion of your retina, plays a role in your central vision; this is required for reading, facial recognition,…
Currently, there are no approved treatment options for myotonic dystrophy type 1 (DM1), a progressive neuromuscular disease. As the disease progresses, affected individuals experience muscle weakness, respiratory distress, and cardiac…
Type 1 narcolepsy, formerly known as narcolepsy with cataplexy, is a chronic neurological disorder characterized by excessive daytime sleepiness and cataplexy, or the sudden loss of muscle tone that is…
Made with fresh watermelon juice, vodka, triple sec, and lemon-lime soda, a Watermelon Crush is exactly as bubbly, bright, and refreshing as you want from your ideal summer sipper. And…
Biopharmaceutical company Inozyme Pharma ("Inozyme") has become a leader at the forefront of developing innovative therapies for rare diseases such as ENPP1 deficiency (the childhood form is known as generalized…
May 2 is Curly Hair Day—a day to celebrate the beauty of waves, coils, and curls. But for one fifth-grader in Canada, Curly Hair Day stands as a way for…
In the United States, Orphan Drug designation comes with benefits and incentives such as fee waivers, tax credits, and up to seven years of market exclusivity if/when the drug is…
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.…
It has been over four years since our lives were irreparably changed by the onset of the COVID-19 pandemic. With hundreds of millions of cases globally, and millions of associated…
In 2010, K. Pattie Figueroa of the Spencer Fox Eccles School of Medicine at University of Utah was approached by a family whose child had spinocerebellar ataxia type 4 (SCA4).…
Clinical-stage biotechnology company Abeona Therapeutics ("Abeona") is working to develop a novel cell therapy for people living with recessive dystrophic epidermolysis bullosa (RDEB), a rare genetic skin disorder characterized by…
For people with geographic atrophy, also known as advanced dry age-related macular degeneration, slowing vision loss is at the top of the list in terms of goals. Treatments like SYOFOVRE…
On December 13, 1996, Health Canada issued a policy statement called Priority Review of Drug Submissions which: provided for the "fast-tracking" of eligible New Drug Submissions (NDS) and Supplemental New…
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.…
Zeposia (ozanimod), developed by Bristol Myers Squibb, is an orally administered sphingosine 1-phosphate (S1P) receptor that is currently approved for two separate indications: relapsing multiple sclerosis and ulcerative colitis. According…
In April 2024, biotechnology company Vertex Pharmaceuticals ("Vertex") shared via news release that it was advancing its therapeutic candidate inaxaplin (VX-147) into the Phase 3 portion of a Phase 2/3…
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