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.
In the average person, you would expect to see normal to near-optimal low-density lipoprotein (LDL) cholesterol levels sitting at 129 mg/DL or lower; levels under 100 are seen as healthier.…
For the first year and a half of her life, Audrey seemed to be a healthy child. Her parents Tenneil and Tom had no real concerns about her health. So…
When biopharmaceutical company Invex Therapeutics (“Invex”) first decided to launch a clinical trial to evaluate Presendin (sustained-release exenatide) for idiopathic intracranial hypertension (IIH), the company felt excited. Presendin is a…
Not everybody understands 9-year-old Michael Martinez’s love for the police. For as long as he can remember, Martinez, who has cerebral palsy (CP), has been fascinated by law enforcement: the…
An estimated 25% of people in the United States have a condition called nonalcoholic fatty liver disease (NAFLD), a condition characterized by excess fat in the liver. Estimates vary but…
Treatment options such as Haegarda and Takhzyro have significantly improved the treatment landscape for people living with hereditary angioedema (HAE). The treatments, used as prophylaxis, work to reduce disease burden…
Orphan Drug designation is granted to therapies that are designed to treat, diagnose, or prevent rare diseases or conditions. In the United States, a rare condition is one affecting fewer…
Nobody expects their child to be diagnosed with cancer. The idea of facing cancer at a young age can be shocking, terrifying. So when 4-year-old Ash Timmerman seemed slightly "off,"…
Raising awareness for rare diseases and conditions is important for many reasons. First, it helps to increase education and understanding around these rare conditions. Awareness also offers support to those…
Caleb Castle-Parker was born ten weeks premature. At the time, his parents didn’t know how his prematurity might affect him (if at all) long-term. But Caleb seemed to be…
In late June 2023, global biopharmaceutical company UCB announced that the U.S. Food and Drug Administration (FDA) approved RYSTIGGO (rozanolixizumab-noli) for adults living with AchR or MuSK antibody positive…
Since 2021, biotechnology company Foghorn Therapeutics ("Foghorn") has been developing its therapeutic candidate FHD-286 for a number of malignancies. In one Phase 1 study, researchers are testing the pharmacokinetics,…
Roivant Sciences recently shared updates on the Phase 2b TUSCANY-2 study. Within the study, the team was evaluating RVT-3101 for moderate-to-severe ulcerative colitis. RVT-3101 is a fully human monoclonal…
13-year-old Kaydence Bryant has always had a big personality. This easygoing teen is an honor roll student, loves playing Pokemon and Skyrim, and plays trumpet in the school band.…
Our genes dictate our health in many ways. When working properly, genes encode for the production of various proteins and enzymes throughout the body. So when genes become mutated,…
Do mast cells—a type of white blood cell that plays a part in immune and allergic responses—contribute to the development of eosinophilic esophagitis (EoE)? In the past, eosinophils (another…
For the first time ever, people living with severe hemophilia A will have access to a gene therapy solution designed to treat their condition at the source. In a…
There are at least 15 different types of dysautonomia, conditions characterized by autonomic nervous system malfunction. Normally, the autonomic nervous system plays a role in digestion, temperature control, blood pressure…
Aboard the private yacht, the crew and passengers delighted in the sights of the open water. It was relaxing onboard: a welcomed break from the pressures of daily life. The…
In August 2022, Payton Quaife’s doctor recommended running some blood tests. The doctor didn’t want to frighten the family. But Quaife had a number of bruises that were healing poorly…
It is important to improve the treatment landscape for various types of cancer. Currently, there is a wealth of research searching for cancer cures. Until that happens, though, we…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
The Orphan Drug Act of 1983 transformed the rare disease treatment landscape. Prior to the Act’s passage, there was little commercial investment in drug development targeted towards rare conditions.…
Right now, there are no available therapies for people living with Fragile X syndrome (FXS). However, clinical-stage biotechnology company Connecta Therapeutics (“Connecta”) is working to change that. In a recent…
“I never thought that it could happen to me.” Unfortunately, we tend to repeat this refrain when facing unexpected hardships in our life from trauma to illness. For many people,…
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