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.
CureDuchenne, a global leader in research and patient care for Duchenne muscular dystrophy (DMD), has been working to find and fund a cure for DMD for more than two decades.…
Continue ReadingKnowledge is Power: CureDuchenne Cares Educational Events Aim to Equip Families and Caregivers in the Becker and Duchenne Muscular Dystrophy Communities
For the first six months of his life, Miller Gamberi seemed to be a happy, healthy boy. But his health suddenly shifted; Miller stopped wanting to eat and drink. His…
Continue ReadingBoy with MHC Class II Deficiency Successfully Treated with Bone Marrow Transplant
Type 1 diabetes (T1D), rheumatoid arthritis, lupus, and ulcerative colitis are all examples of autoimmune diseases. An autoimmune disease is a condition that occurs when the immune system mistakenly attacks…
Continue ReadingStudy Suggests that Perinatal Depression Risk is Significantly Higher in Women with Autoimmune Diseases
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.…
Continue ReadingRare Community Profiles: How Matrix Can Be Leveraged to Co-Create and Co-Deliver Care in the Rare Disease Space
Ring chromosome 14 syndrome is an extremely rare chromosomal disorder that was first described in medical literature in 1971. An estimated 40-80 cases have since been discovered and described. So…
Continue ReadingCharlieFest on March 9: An Event to Raise Ring Chromosome 14 Syndrome Awareness
An estimated one in every four women and one in every nine men is a victim of domestic violence, writes Huecker, King, Jordan, and Smock (2023). This accounts for about…
Continue ReadingUSC Alumna Raises Awareness of Relationship Between CTE and Domestic Violence
Genes serve as the intricate orchestrators of our health. By better understanding our genetic makeup, we gain invaluable insights into how our genes shape and affect us. Yet the genetic…
Continue ReadingNaturally Occurring SHLP2 Variant Protects Against Parkinson’s Disease
Chronic spontaneous urticaria (hives) - an itch that can't be scratched. Well, from a literal perspective, you can scratch. But the chronic nature of chronic spontaneous urticaria means that your hives will…
Continue ReadingPhase 1 Clinical Study Initiated to Evaluate EVO756 for Chronic Spontaneous Urticaria
Immunotherapy, targeted therapy, surgery, radiation, chemotherapy, intravesical treatment - oh my! These therapeutic options have all been used to treat individuals living with urothelial carcinoma. But the treatment landscape has…
Continue ReadingFDA Approves Balversa (Erdafitinib) for Locally Advanced or Metastatic Urothelial Carcinoma
Editor's Note: Chronic conditions and rare diseases don't discriminate, Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have…
Continue ReadingCould Low Serotonin Cause MCI and Alzheimer’s Disease?
Editor's Note: Chronic conditions and rare diseases don't discriminate, Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have…
Continue ReadingHad High BMI as a Child? Your Risk of Chronic Kidney Disease (CKD) in Adulthood May Be Heightened
Editor's Note: Chronic conditions and rare diseases don't discriminate, Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have…
Continue ReadingFLORetina-ICOOR Meeting: Do GLP-1 Agonists Increase Risk of Diabetic Retinopathy?
In December 2023, clinical-stage biopharmaceutical company Savara Inc. announced that the company had launched a serum-based blood test to aid in diagnosing autoimmune pulmonary alveolar proteinosis (aPAP). While aPAP is…
Genes hold the key to our health. Through revolutionary gene therapy solutions, the medical field is transforming to laud the power and precision of personalized medicine. Gene therapy has the…
Continue ReadingUMass Chan Progressing on Gene Therapy Vector for Cockayne Syndrome
Photo courtesy of Greg Hadley and Uplifting Athletes
In the hallowed halls of Colgate University, a young man named Greg Hadley found his calling in an unexpected place: the field of rare diseases. Little did he know that…
Continue ReadingFriends in the Fight: Athlete’s Loss Inspires Support for Tay-Sachs Research via Uplifting Athletes’ Young Investigator Draft
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.…
Continue ReadingRare Community Profiles: Peggy Lillis’ Legacy: How the End of an Extraordinary Life Launched a Foundation for C. Diff Education, Awareness, and Policy
In December 2023, pharmaceutical and biotechnology company Moderna announced that results from a Phase 2/3 clinical trial were published in The New England Journal of Medicine. The study explored an mRNA-based…
Continue ReadingICYMI: Phase 3 Results on mRNA-1345 for RSV Published in NEJM
For the first six months of his life, Oliver Mills seemed to be developing fairly normally. He was a happy, bubbly baby. His mother Laurel found joy in every smile,…
Continue ReadingOliver’s Story: What It’s Like to Live with Beta-Mannosidosis
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.…
Continue ReadingRare Community Profiles: A Conversation with Rallybio CEO Stephen Uden on the Need for Rare Disease Drug Development
Omega-3 fatty acids like alpha-linlenic acid or docosahexaenoic acid are "healthy fats" that are found in foods like flaxseed, fish, soybeans, and canola oil; they may also be given via…
In December 2023, clinical-stage biopharmaceutical company Clene Nanomedicine ("Clene") shared that it had met with the U.S. Food and Drug Administration (FDA) regarding Accelerated Approval for CNM-Au8®, an experimental therapy…
Continue ReadingNo Accelerated Approval for Experimental ALS Drug CNM-Au8
The research landscape for Duchenne muscular dystrophy (DMD) has been steadily increasing. Researchers are exploring more therapies, including gene therapies, that could transform the lives of those with this rare…
Continue ReadingSGT-003 Granted Orphan Drug Designation for Duchenne Muscular Dystrophy (DMD)
In 2015, the U.S. Food and Drug Administration (FDA) approved NUCALA (mepolizumab) for the treatment of severe eosinophilic asthma in adolescents and adults ages 12 and older, and by 2022,…
Continue ReadingNUCALA Now Approved in China for Severe Eosinophilic Asthma
In mid-January 2023, Kristi Rosa of OncLive reported that the European Medicines Agency (EMA) granted Orphan Drug designation to pimicotinib (ABSK021) for inoperable tenosynovial giant cell tumors (TGCT). Orphan Drug…
Continue ReadingPimicotinib Earns Orphan Drug Designation from the EMA for Tenosynovial Giant Cell Tumors
Do you have cystic fibrosis? Are you searching for a stronger sense of community? Then BreatheCon, the unique virtual event spearheaded by the Cystic Fibrosis Foundation, is perfect for you! …
Continue ReadingCystic Fibrosis Foundation to Host 8th Annual BreatheCon on February 9-10, 2024
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