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…
When Brianna Terry was just two years old, her doctors gave her a diagnosis of autosomal recessive osteopetrosis. At the time, the doctors were concerned about her wellness; they…
In the United States, the FDA grants Orphan Drug designation to drugs or biologics intended to treat, diagnose, or prevent rare conditions. A rare disease or condition is defined as…
Data from a Phase 2 trial examining BI 10115550, an oral treatment for idiopathic pulmonary fibrosis (IPF), has just been released. This data shows that this therapy is able to…
According to a story from the BHD Foundation, Patient Worthy partner The Myrovlytis Trust, along with the BHD Foundation and Pulse Infoframe, are collaborating on an international Birt-Hogg-Dubé (BHD) syndrome…
In the past, there have been a variety of research studies exploring and working to learn more about cystinuria, a rare genetic condition causing cystine, an amino acid, to accumulate…
Sometimes, it can be difficult to prompt the research of and creation of therapeutics for rare diseases. This is one of the reasons why the FDA created its Orphan Drug…
Every year, people from across the world come together to celebrate Myasthenia Gravis Awareness Month during the month of June. The Myasthenia Gravis Foundation of America (MGFA) plays a big…
In 2022, the 25th Annual Meeting of the American Society of Gene & Cell Therapy took place from May 16th-19th. During the meeting, researchers and other stakeholders discussed the latest…
Two years ago, in 2020, life changed for Ian Foster, then 57. At the time, Ian started experiencing health problems and was eventually diagnosed with kidney failure. According to The…
Medscape recently published an article stating that the EMA is conducting a review of rucaparib, a drug used to treat patients with a range of ovarian cancers that have relapsed. These…
Chinese patients living with pediatric growth hormone deficiency are one step closer to a new treatment option, according to a recent article from PharmaBiz.com. The step I'm referring to is…
According to a story from Scleroderma News, June is recognized as Scleroderma Awareness Month, a time for spreading awareness about scleroderma among the general public and in the medical field.…
Written by: Tyler Bradley, PFIC Network The PFIC Scientific Conference Held Many Great Presentations and Roundtables That Informed Both The Scientific And Patient Communities. Last month the PFIC Network hosted…
According to a recent article, scientists were able to create cilia-free stem cells that may help in finding the causes of cilia-linked illnesses such as polycystic kidney disease. …
Acromegaly is a rare disease that is characterized by excess and abnormal levels of growth hormone (GH). Because of this, medical professionals are very interested in these levels as patients…
Nalla Lawson, who lives in Ocala, Florida, recently shared her story and treatment journey through an article published in UF Health. After suffering a fall back in 2016, she was…
According to a recent article, various tick species carrying dangerous pathogens are spreading across the United States, making it more important to know how to keep yourself safe. Tick-Borne Diseases…
Data based on approximately five million people suggests that esophageal cancer has nearly doubled from 2012 through 2019. According to a recent report in MSN, the incidence of precancerous Barrett’s…
Regeneron Pharmaceuticals Inc. has just released positive results from their Phase 3 investigation of Evkeeza as a treatment for homozygous familial hypercholesterolemia (HOFH). Specifically, this trial studied the treatment for…
It’s no secret that living with a rare or underserved condition can come alongside a heavy financial burden. A recent study supported by the National Institutes of Health (NIH), Cure…
CureDuchenne is a nonprofit whose mission is to find a cure for Duchenne muscular dystrophy (DMD), a rare progressive disease characterized by muscular degeneration. It is diagnosed in one individual…
PTC Therapeutics Inc. has just announced that their gene therapy called Upstaza has received a positive opinion from the EMA. After the therapy is ratified by the European Commission (EC),…
On May 26, 2022, biopharmaceutical company Nurix Therapeutics, Inc. ("Nurix") shared via news release that positive data was available from an ongoing Phase 1a dose escalation study. Within the study,…
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…
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