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.
Prior to launching a clinical trial, the FDA must clear an Investigational New Drug (IND) application. This allows the drug to be distributed across state lines, which is important…
Continue ReadingFDA Clears Clinical Study to Evaluate BB-301 for Oculopharyngeal Muscular Dystrophy
Even with available therapies, pancreatic cancer is incredibly tough to treat and comes with a poor prognosis. Only 12% of people diagnosed with pancreatic cancer are still alive after…
Continue ReadingElraglusib Earns Orphan Drug Status for Pancreatic Cancer
In a late July 2023 news release, global healthcare company Grifols shared that positive topline data was now available from a Phase 4 clinical study evaluating XEMBIFY (immune globulin…
Continue ReadingPositive Data Available from Phase 4 Trial for Primary Immunodeficiencies
Between 2019 and 2020, the number of reported Hansen’s disease (leprosy) cases across the United States fell. However, there have been an uptick of cases since 2020, especially in…
Continue ReadingCould Hansen’s Disease Become Endemic in Florida?
For as long as she can remember, eighth-grader Ella Bork has held a keen knowledge of rare diseases, especially Alagille syndrome. Her mother, Cher, is actually the Executive Director of…
Continue ReadingEighth Grader Ella Has Raised $5K for Alagille Syndrome Research and Support
65 roses. This phrase is often used in reference to cystic fibrosis; in fact, the origin of 65 roses is a young boy who couldn’t properly pronounce the name…
Continue Reading“Make That Geezer Get Those Tattoos:” A Cystic Fibrosis Fundraising Mission May Result in 65 Tattoos
Each year, there are less than 600 new cases of papillary craniopharyngioma (PCP) diagnosed within the United States. While PCP typically has a good prognosis with treatment, treatment is…
Continue ReadingCould Melanoma Drugs be Effective in Treating Papillary Craniopharyngioma (PCP)?
First established by the International Foundation for Gastrointestinal Disorders (IFFGD) in 2016, Gastroparesis Awareness Month is designed to increase awareness of gastroparesis and how it affects the people who…
Debbie Ferlito had always lived an active life. There was nothing she loved more than throwing herself into athletics, than testing the limits of her body. And she was…
Continue ReadingHer Balance Issues Eventually Led to Her RFC1 CANVAS Diagnosis
In early June 2023, a Kansas City Royals’ right-hand pitcher (RHP) named Josh Staumont was placed on the injured list (IL) due to a neck strain. While he was…
Continue ReadingRoyals’ Josh Staumont on Will Undergo Thoracic Outlet Syndrome Surgery
A Phase 2 clinical study set out to determine whether prexigebersen, alongside decitabine and venetoclax, would be effective in treating and stopping the progression of a rare cancer of…
Continue ReadingAn Interim Data Highlights the Potential Benefits of Prexigebersen for AML
In 2020, the Denver Broncos drafted wide receiver K.J. Hamler. Thus far in his career, Hamler has registered 42 receptions, 620 receiving yards, 3 touchdowns, and 24 receiving first…
At five years old, Traviana Dunston has cultivated a deep love and appreciation for music and dance. She’s an entertainer at heart: full of energy and light. Right now, Traviana…
Continue ReadingMom Raises Awareness After Daughter’s DIPG Diagnosis
Orphan drug designation was first introduced to the European Union in 2000. The designation is granted to therapies intended to treat, diagnose, or prevent a rare, life-threatening, or chronically…
Continue ReadingTaldefgrobep Alfa Earns EU Orphan Drug Status for Spinal Muscular Atrophy (SMA)
Have you ever heard of Orphan Drug designation? This special status was created to incentivize the development of therapies for people with rare conditions (affecting fewer than 200,000 people…
Continue ReadingTYRA-300 Snags Orphan Drug Designation for Achondroplasia
It can be difficult to grasp the scope of a rare disease, its symptoms, and impact—especially if certain individuals don’t fall within the understood or narrowly defined parameters of…
Continue ReadingICYMI: Updated Diagnostic Criteria Available for Kleine-Levin Syndrome (KLS)
In a Phase 1 clinical study, researchers wanted to understand whether ALTB-268, developed by clinical-stage biotechnology company AltruBio Inc., could be effective in treating ulcerative colitis. The study first…
Continue ReadingPositive Topline Data Available on ALTB-268 for Ulcerative Colitis
Hudson Reynolds learned about rare disease when his sister Tia was diagnosed with phenylketonuria (PKU). PKU is an inborn error of metabolism which causes phenylalanine, an amino acid, to build…
Continue ReadingTeen Climbs Mt. Kilimanjaro to Raise PKU Awareness
Technology is everywhere, pervasive in our day-to-day lives. We get our news from technology, work using technology, and connect with our community via technology. Advances in this field are also…
Continue Reading“Tech Neck:” How Too Much Screen Time Can Cause Thoracic Outlet syndrome (TOS)
Earlier this year, the FDA granted Accelerated Approval to ELEVIDYS (delandistrogene moxeparvovec-rokl) for the treatmnt of Duchenne muscular dystrophy (DMD) in boys aged 4-5. While this is a stunning…
If gastric cancer is localized (has not yet spread), it carries a significantly higher 5-year survival rate of 72-75%. But once the cancer spreads, the rate falls lower: 35%…
Continue ReadingZolbetuximab Under Priority Review for HER2-Negative Gastric Cancer
People in China who are living with narcolepsy will soon have access to a new therapeutic option to help them manage cataplexy or excessive daytime sleepiness (EDS) after the…
Continue ReadingPitolisant Now Approved in China to Treat Excessive Daytime Sleepiness or Cataplexy in Narcolepsy
The 25th World Congress of Dermatology took place this year from July 3 to 8, 2023. During the Congress, stakeholders throughout the medical industry convened to discuss research, trends, and…
Two days. That’s all it took for Jill and Jeff Ackermann to watch their son’s condition mysteriously deteriorate. On Saturday, six-year-old Bryson was the energetic, athletic, and bright-eyed little boy…
Continue ReadingHe Battled Acute Flaccid Myelitis (AFM)—And Now He’s Back to Playing Baseball!
Having a rare or uncommon condition can be undoubtedly overwhelming. The diagnosis comes with associated mental health, financial, physical, and social burdens that can weigh heavy on your mind. But…
Continue ReadingEurope Approves Columvi for Relapsed or Refractory DLBCL
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