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: A Mother-Son Pair Discuss the Importance of Wellbeing in Managing NF2-Related Schwannomatosis (NF2)
Pulmonary arterial hypertension (PAH) is a rare, chronic, and progressive disorder characterized by high blood pressure in the lungs. This causes pulmonary arteries to thicken or harden, which eventually puts…
Continue ReadingWinrevair is Now FDA-Approved for Pulmonary Arterial Hypertension (PAH)
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: Yellow for Yiannis: Angela’s Mission to Advance Research into IRF2BPL
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 Mother’s Journey to Raise 4H Leukodystrophy POLR3B Awareness and Change Her Son’s Life
Historically, it has been difficult to incentivize companies to develop therapies for rare or “orphan” conditions. The National Conference of State Legislatures (NCSL) explains that “orphan” conditions are: neglected conditions…
Continue ReadingICYMI: AIT-101 Earned Orphan Drug Designation in the EU for ALS in March 2024
There are limited therapeutic interventions for those living with Niemann-Pick disease type C (NPC), a rare and progressive genetic disorder. Unfortunately, many people with NPC experience severe and life-threatening complications…
Continue ReadingPhase 2 Study Evaluates Nizubaglustat for Niemann-Pick Disease type C
The Duchenne muscular dystrophy (DMD) community has a reason to celebrate. According to a recent news release, the FDA recently approved Duvyzat (givinostat) for people ages six and older who…
Continue ReadingDuvyzat (givinostat) Now FDA-Approved for Duchenne Muscular Dystrophy (DMD)
Ronald Acuña Jr. is a professional baseball player with the Atlanta Braves. The powerhouse player is known for being an NL MVP and for becoming the first player in Major…
Continue ReadingAtlanta Braves’ Ronald Acuña Jr. Plays Catch with Fan Living with Cystic Fibrosis (CF)
There are over 30 subtypes of congenital muscular dystrophy (CMD), or muscular dystrophies that are present at birth. These rare genetic disorders often manifest in hypotonia (low muscle tone) and…
Continue Reading“Rockin’ for Rudy” Supports a Family Whose Son Has LAMA2-MD
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: Could It Be HCM? A Mother-Daughter Duo Raise Hypertrophic Cardiomyopathy Awareness
People with Cushing's disease have a benign pituitary tumor or pituitary growth that, when triggered, overproduced adrenocorticotropic hormone (ACTH). Too much ACTH causes the body to also overproduce cortisol. Known as…
Continue ReadingAchieving Cushing’s Disease Remission After Surgery Could Increase Risk of New-Onset Autoimmune Disease
Dupixent has long been known as a therapy which reduces itch, slows disease progression, and reduces exacerbations in a number of allergic conditions such as asthma, eczema, and nasal polyps.…
Continue ReadingPovorcitinib Performs Well in Phase 2 Prurigo Nodularis Study
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 Potential Treatment Turning Point for ALK-Positive NSCLC: Dr. Ken Culver Shares Insights from the Phase 3 ALINA Study on Alecensa
Parkinson's disease is a progressive neurodegenerative disease. As neurons in the brain degenerate and die, those affected experience symptoms such as tremors or shaking in the hands, slowed movement, muscle…
Continue ReadingPositive Data Suggests NT-0796 Could Benefit People with Parkinson’s Disease
Right now, no cure exists for people living with systemic sclerosis (also known as systemic scleroderma). There are treatment options available to manage systems. However, these also come with issues.…
Continue ReadingCABA-201 for Systemic Sclerosis Earns Orphan Drug Designation
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: For Rare Disease Week, Barth Syndrome Advocates Took to Capitol Hill to Urge the FDA to Review NDA for Elamipretide
The Sudden Arrhythmia Death Syndromes (SADS) Foundation - a Patient Worthy partner - was founded in 1991 to provide support and care for people affected by heart arrhytmia conditions that…
Continue ReadingSADS Foundation Will Meet with FDA in June on New Treatments for LQTS and CPVT
In the world of sports, the spotlight often shines on athletic prowess and competitive spirit. Yet Uplifting Athletes—a Patient Worthy partner—has transformed the narrative on the power of sport for…
In early March 2024, Tyler Patchen reported that the U.S. Food and Drug Administration (FDA) approved both intravenous and subcutaneous formulations of Tyenne (tocilizumab-aazg) for use in a variety of…
Continue ReadingFDA Approves Tyenne, an Actemra Biosimilar for Autoinflammatory Diseases
Since 2015, the National Organization for Rare Disorders (NORD) has released an annual State Report Card that highlights state policies which could affect how people with rare diseases access care.…
Continue ReadingNORD State Report Card: Each State Graded on Rare Disease Care Access
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 Patient Advocate Kecia J. Survived and Thrived Through Her Rare Colorectal Cancer Battle
A majority of available treatment options for metachromatic leukodystrophy (MLD) rely on relieving symptoms. However, the recent approval of Lenmeldy (atidarsagene autotemcel) is the first ever FDA-approved gene therapy option…
Continue ReadingFDA Approves Gene Therapy Lenmeldy for Metachromatic Leukodystrophy (MLD)
When Josh Wright, 13, was younger, he always seemed to be somewhat of a sickly child. His mother Julie remembers Josh's first hospitalization at just one year old; her…
Continue ReadingLiving with Cyclic Vomiting Syndrome: Josh’s Story
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: In Pursuit of Progress: From HSP Diagnosis to Rare Disease Research
On March 7, 2024, the United Kingdom’s Medicines and Healthcare products Regulatory Agency shared via press release that the agency has approved Ztalmy (ganaxolone) for people living with CDKL5 deficiency…
Continue ReadingUK Approves Ztalmy (Ganaxolone) for CDKL5 Deficiency Disorder
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