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.
Charcot-Marie-Tooth disease (CMT), though rare, is one of the most commonly inherited neurological disorders and affects peripheral nerves outside of the brain and spinal cord. Multiple CMT subtypes exist based…
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CMT Research Foundation Invests in Study Evaluating Potential Therapy for CMTX1
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…
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Brothers Are One of Only Two Known Sibling Pairs with ROHHAD
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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…
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One Woman’s Journey to Raise Cushing Disease Awareness
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…
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RETINA WORLD CONGRESS: Diabetic Macular Edema Clinical Trials Lack Patient Diversity
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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…
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Antibiotic Exposure in Infancy May Increase the Risk of Developing Atopic Dermatitis
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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…
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Volleyball Tournament Supports Boy with Apert Syndrome
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)…
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Wild Wickenburg Bull Riding Fundraising Raises $5K for Teen with MPGN II
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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…
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May 2024: Celebrating Vasculitis Awareness Month!
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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…
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Spinogenix’s SPG601 Earns Orphan Drug Designation for Fragile X Syndrome
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,…
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Metastatic Breast Cancer: Navigating Grief
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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,…
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First Patient Dosed in Clinical Study Evaluating Kamuvadine-8 for Geographic Atrophy
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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…
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AOC 1001 Earns Breakthrough Therapy Designation for Myotonic Dystrophy Type 1 (DM1)
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…
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Phase 2 Trial Commences for ALKS 2680, a Potential Game-Changer for Type 1 Narcolepsy
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…
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Drink a Watermelon Crush in PA This Summer to Support People with Rare Diseases
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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…
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ICYMI: INZ-701 Begets Positive Data in Clinical Study on ABCC6 Deficiency and ENPP1 Deficiency
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…
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Boy with Giant Axonal Neuropathy (GAN) Celebrates Curly Hair Day at School
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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…
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Wilate Granted Orphan Drug Exclusivity for Severe Von Willebrand Disease
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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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Rare Community Profiles: From Survivor to Advocate: Ethan Zohn on Cannabis Rescheduling and Cancer Care
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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…
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Triathlete Describes Damaging Long-Term Impact of Long COVID
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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).…
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Researchers Identify How GCC-Repeat Expansion in ZFHX3 Causes Spinocerebellar Ataxia Type 4
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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…
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FDA Rejects Epidermolysis Bullosa Cell Therapy and Requests Additional Manufacturing Data
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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…
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Dosing Complete in OCU410 Trial for Geographic Atrophy
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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…
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ICYMI: Exa-cel for Beta Thalassemia, Sickle Cell Disease Accepted for Priority Review by Health Canada
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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Rare Community Profiles: How Two Parents Are Working to Change the Narrative Around CASK Gene Disorder
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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…
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Zeposia Trial for Crohn’s Disease Falls Short