In a story from BioPharma Dive, a number of high-profile clinical trials are under way that could see conclusive results by the end of 2023. While some of these are…
The incidence of gastroschisis has doubled over the past 20 years. Read “Jeanie’s Gastroschisis Baby” and learn from a brave, young first-time mom about discovery and how Gastroschisis can be…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
In the past, doctors have noticed a trend in hepatocellular carcinoma (HCC). More specifically, research has shown that HCC can grow resistant to certain drugs. This makes it more…
Dr. William Lynes completed his residency in 1987 at Stanford University. He began his practice at the Kaiser Permanente Riverside Medical Group in California. It was the idyllic life that…
The American Society for Clinical Oncology (ASCO) held its Annual Meeting in late May and early June to discuss clinical guidelines, best practices, and research advancements within the oncology…
The Orphan Drug Act of 1983 was implemented after the U.S. Food and Drug Administration observed drug development issues in the rare disease space. Rare diseases often have smaller…
Both Ofev (nintedanib) and Esbriet (pirfenidone) are FDA-approved to treat individuals living with mild, moderate, or severe idiopathic pulmonary fibrosis (IPF). The treatments work by preventing fibrosis (scarring) and stopping…
When it comes to advancing treatments and research, collaboration is key. A new partnership recently formed; this collaborative effort underscores the need for additional support and resources for people living…
On June 21, 2023, the Rare Disease Legislative Advocates (RDLA) hosted its monthly webinar. These webinars help provide updates to the rare disease community on legislation and other policy initiatives…
It looked as if the fate of Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy was pretty much sealed until an FDA official intervened. This information was provided by…
As she stood in the garden, surrounded by friends and family, a splitting headache richocheted through Ciara Wilkie's head. Her vision blurred. At first, Ciara didn't think much of it.…
When it comes to treating interstitial cystitis, doctors may take a multipronged approach: physical therapy, bladder distention, surgery, nerve stimulation, oral medications. But people with this condition lack therapies…
The U.S. Food and Drug Administration offers various designations to drugs and drug developers to both incentivize medical advancements in the rare disease space and get treatments in the…
In 2015, rare disease stakeholders in North Carolina established the first Rare Disease Advisory Council (RDAC); the National Organization for Rare Disorders (NORD) explains that an RDAC is an…
As recently reported by Forbes, the U.S. Food and Drug Administration (FDA) approved Leqembi (lecanemab) for Alzheimer’s disease earlier this week. Leqembi is a humanized monoclonal antibody that targets…
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…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
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,"…
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