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.
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: When Sierra’s Son Was Diagnosed with Warsaw-Breakage Syndrome (WABS), She Knew She Needed to Make a Difference
In the United States, a rare condition is defined as one affecting fewer than 200,000 people nationwide. But when you consider how many people actually live with a rare condition,…
Continue ReadingMississippi Has Established a Rare Disease Advisory Council (RDAC)
What’s better than being in warm and sunny Arizona? Getting to learn alongside the amazing primary biliary cholangitis (PBC) community while there! The PBCers organization held its 2024 Patient Education…
Continue ReadingRECAP: My Time at the PBCers 2024 Patient Education Conference: “Empowering Connections in PBC”
Developing an animal model of a disease can play an important role in both helping researchers to better understand that disease, as well as identifying and testing potential therapeutics. Animal…
Continue ReadingTMDU Researchers Develop Mouse Model for Anti-MDA5 Antibody-Positive Dermatomyositis
In September 2023, the Virginia Department of Health announced that five people across the state died from a rare, serious, and somewhat unusual strain of meningococcal disease. Now, reports Brenda…
Continue ReadingRare Meningococcal Disease May Present with Unique Symptoms
There are a number of symptoms and characteristics associated with Kindler epidermolysis bullosa (Kindler EB), a rare genetic disorder. When researchers wanted to understand whether hypoplastic pitted amelogenesis imperfecta (AI),…
Continue ReadingKindler Epidermolysis Bullosa Characterized by Oral and Dental Abnormalities
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 a Clinical Study Changed Dana’s Trajectory After Desmoid Tumor Recurrence
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 Kenzi is Navigating the Challenges of MCTD, FCAS, and Medical PTSD
Lupus nephritis is a complication of lupus that refers to the kidney inflammation and damage that can occur. In lupus nephritis, the affected individual's immune system mistakenly attacks the kidneys,…
Continue ReadingFirst Patient Dosed in Study Evaluating AlloNK (AB-101) for Lupus Nephritis
Medication can be an essential component of managing attention deficit hyperactivity disorder (ADHD). These medications, such as Vyvanse and Adderall, can help to: Reduce symptoms like impulsivity, hyperactivity, and inattention…
Continue ReadingADHD Drug Shortages Mean People Aren’t Getting Their Meds
In some cases, healthcare providers can identify Angelman syndrome in utero through a prenatal ultrasound or prenatal genetic testing. But could this open the door to early treatment and better…
Continue ReadingCould We Treat Angelman Syndrome and Other Genetic Disorders in Utero?
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: Revving for Hope: Raising HoFH Awareness in One Ride
Ultomiris (ravulizumab) and Soliris (eculizumab), both developed by global biopharmaceutical leader AstraZeneca, are both effective treatments for paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). Yet this efficacy…
Continue ReadingDanicopan Approved as Add-On Therapy for PNH Treatment
When you’re a baseball fan, any chance you get to join in and really experience the game is amazing. Seven-year-old Ella McKee has always been fascinated by baseball. She loves…
Continue ReadingRaising Vanishing White Matter Disease (VWM) Awareness: How Ella’s Pitch Catalyzed Change
Vision loss can be challenging. If you could have a one-time gene therapy that would improve your visual acuity, would you take it? It seems like a no-brainer. That's what…
Continue ReadingOne-Time Gene Therapy ABBV-RGX-314 Shows Potential in Wet AMD
Researchers from the Max Planck Institute for Evolutionary Anthropology (MPI-EVA) have been collecting and analyzing data on ancient DNA for years. This DNA has given the researchers a stronger…
Continue ReadingEdwards Syndrome and Down Syndrome Identified in Ancient DNA
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 Beth and Madison Advocated for Public Funding for the First Cystic Fibrosis (CF) Modulator in Canada
Anna-Lena Neehus of the Imagine Institute at Paris Cité University was searching through genomic data from over 15,000 patients. She wanted to understand how and whether genetic deficiencies contributed to…
Continue ReadingICYMI: Researchers Discover New Genetic Disorder When Linking CCR2 Deficiency to Pulmonary Alveolar Proteinosis
The European Society of Cataract & Refractive Surgeons (ESCRS) held its ESCRS Winter Meeting from February 15-18, 2024. During the meeting, Dr. M.S. Swathi, MD, shared data from a pilot…
Continue ReadingIntrastromal Lamellar Keratoplasty with Collagen Cross-Linking Improves Progressive Keratoconus
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: The Urgent Need for a Cure for Niemann-Pick Type C: Two Parents Share Their Perspectives
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: Kristi’s Fight to Combat Misconceptions about Prader-Willi Syndrome (PWS): “Our Children Have PWS; They Are Not PWS”
April is Sarcoidosis Awareness Month. During this month, people with sarcoidosis, caregivers, physicians, and other supporters come together to amplify the stories of those affected, reinforce the importance of continued…
Continue ReadingCyclist Shares Her Story During Sarcoidosis Awareness Month
While there is no cure for myasthenia gravis, this disorder can be managed with treatment. Current treatment options include a thymectomy, C5 protein inhibitors, and intravenous immune globulins (among others).…
Continue ReadingEnspryng Falls Short in Generalized Myasthenia Gravis Clinical Study
Over the past few years, Akero Therapeutics has experienced high highs and low lows in its efforts to advance efruxifermin, an investigational drug for people living with metabolic dysfunction-associated steatohepatitis…
Continue ReadingUpdated Study Data Suggests Efruxifermin Benefits Stage 2 or 3 MASH
Pulmonary arterial hypertension (PAH), a rare and chronic disorder characterized by high blood pressure (hypertension) in the lungs that causes the pulmonary arteries to thicken or harden, most often manifests…
Continue ReadingOpsynvi Now Approved for Pulmonary Arterial Hypertension (PAH)
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