If your child was sick and there was a real, viable treatment option, the next step seems pretty straightforward: pursuing that option. But what if it wasn’t that easy? What…
According to a news release, researchers from biopharmaceutical company HUTCHMED and pharmaceutical company AstraZeneca launched, at the onset of November 2021, the Phase 3 SAMETA clinical trial. During the trial,…
In Part 1 of Rachael's story, I spoke with her mother Debbie about what Niemann-Pick type C (NPC) is, the beginning of the diagnostic journey, and how Dr. Behar helped…
Over the past few weeks, Centenary College has partnered with Be The Match to host a stem cell donor drive. The first two drives were on November 18th and 19th,…
Since the onset of COVID-19, there have been approximately 262M cases, and 5.2M deaths, on a worldwide scale. However, the introduction of various COVID-19 vaccines, such as those from Pfizer…
In Part 1 of "The TSC Navigator: A New Resource to Guide Families through the TSC Journey," I sat down with Ashley Pounders to discuss the TSC Navigator, its purpose,…
A recent study published in Nature and Science of Sleep looked at asthma and its possible connection to narcolepsy, actually finding indications that an asthma diagnosis raises the risk of…
Childhood cancer is an area that requires research and innovation, as there are currently many unmet medical needs within these patient populations. Fortunately, one non-profit has been working on raising…
Mereo BioPharma Group has just announced that they have received Orphan Drug Designation from the FDA for their new treatment for alpha-1 antitrypsin deficiency (A1AD) called alvelestat. This therapy is…
“Hi, I’m Rachael, and I’m forever 33.” When Debbie Kaflowitz remembers her daughter, she thinks about everything that Rachael loved to do. Rachael enjoyed dancing and performing in ballet recitals,…
A recent study was published in JAMA Oncology outlining a retrospective cohort study analyzing cabozantinib as a treatment for renal cell carcinoma that has metastasized to the brain. According to the data,…
In mid-November 2021, global biopharmaceutical company Zai Lab Limited ("Zai Lab") shared via news release that the first patient was dosed in the Greater China portion of the ADHERE study.…
The TSC Alliance® first began as a grassroots organization, founded by motivated mothers whose children had been diagnosed with tuberous sclerosis complex (TSC), a rare genetic disease. Since its founding,…
Nancy Green and Judi Beckerleg first met nearly 22 years ago in 1999. After Judi moved to Arizona, she began working as an aide in Nancy's classroom, where Nancy taught…
Claire Richmond lives with acute hepatic porphyria (AHP). She searched for 19 years to find answers to her unexplained pain. She wasn't diagnosed until she was 32. She recently had…
Results from a study on systemic mastocytosis (SM) have recently been published. This investigation aimed to understand the prevalence of contrast media induced hypersensitivity reactions (CMIHR) in different subtypes of…
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…
According to a recent article, researchers investigated the cause of deaths in the hospital in patients who were diagnosed with inflammatory myositis in a tertiary care center in Northern…
According to an article published by Alnylam Pharmaceuticals, a recent survey has provided insight into the burden experienced by acute hepatic porphyria (AHP) patients. Titled the POWER (POrphyria Worldwide Patient…
Thanksgiving is a time of gratitude, family and maybe a little gluttony. It was always my favorite time of the year in high school and college. Now, while I still…
When you are looking for new treatments that will either stop or slow the development of Alzheimer’s disease, where would you look? According to a recent article in Gladstone…
At this time last year, I was 89 lbs and at the beginning stages of recovery from a year of IV and oral antibiotics for the treatment of Lyme disease.…
CRISPR is a gene-editing technique that has revolutionized the medical world with its approach to making precise changes in DNA. CRISPR found its match in 35-year-old Victoria Gray of…
According to a recent article, PharmaTher Holdings Ltd. announces the FDA granted orphan drug designation (ODD) for ketamine in the treatment of complex regional pain syndrome (CRPS). Complex Regional…
According to a recent article, mental health affects the physical body in a number of ways, and it’s important to recognize and understand this connection. Mental health has such a…
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