Colorado is a beautiful state for fly fishing, and it attracts many people who love the sport. A group of anglers from this state has joined together, united by both…
The FDA will review parsaclisib as a treatment for Non-Hodgkin lymphoma (NHL) after accepting Incyte's New Drug Application (NDA). Specifically, the NDA was submitted for the treatment of relapsed or…
A recent poster presentation at the 2021 NANETS Annual Meeting focused on the RELAX study, which evaluated patient-reported outcomes from carcinoid syndrome (CS) patients being treated with telotristat ethyl. According…
Jo Traunter was told that she was "a bleeder" throughout her life, whether it was by family, doctors, or others. She earned this title after living through decades of heavy…
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,…
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…
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,…
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…
.png)
GIVLAARI has already received approval for the treatment of acute hepatic porphyria (AHP) in Canada, according to an article from Nasdaq. Now, the Canadian Agency for Drugs and Technologies in…
Recently the Kidney Cancer Association held its International Kidney Cancer Symposium, where medical professionals and other industry members come together to share ideas and push for innovative research and treatments…
Back in June of this year, the FDA granted approval to UPLINZA to reduce attacks in patients with anti-aquaporin-4 immunoglobulin G seropositive (AQP4-IgG+) neuromyelitis optica spectrum disorder (NMOSD). Now, the…
From November 21st until the 23rd, medical professionals and others involved in the metabolic field will come together for the 14th International Congress of Inborn Errors of Metabolism (ICIEM). It…
The COVID-19 vaccine was approved quickly by the FDA in an effort to fight the raging pandemic. Due to the rapid pace of these trials, a number of populations weren't…
November is officially recognized as National Family Caregivers Month, a time dedicated to recognizing and celebrating caregivers. The entire month is full of recognizing the effort and needs of caregivers,…
According to an article published in Yahoo, the FDA has recently granted the Rare Pediatric Disease designation to AVR-RD-05, a gene therapy for Hunter syndrome. This designation is reserved for…
The FDA grants certain designations, such as the Orphan Drug (ODD) and Fast Track designations (FTD), to medications intended to treat rare and severe disorders. Recently, they granted both of…
It is often difficult to estimate the prevalence of a rare disease within the United States. For one, it can be difficult to find all of the patients with a…
Data from the TELEPRO-II study of Xermelo were recently published in the Journal of Cancer Management and Research. It investigated this oral therapy as a treatment for carcinoid syndrome (CS)…
According to a press release from Ikena Oncology, the FDA has recently accepted an Investigational New Drug (IND) application for Ikena's investigational drug candidate, IK-930. This TEAD inhibitor is intended…
When a disease is progressive, it can be difficult to discern if treatment is working. Transthyretin amyloid cardiomyopathy (ATTR-CM) is an example, especially as its treatment, tafamidis, presents "relatively few…
Canavan disease is an ultra-rare condition that currently has no FDA-approved therapies, meaning that patients face a severe unmet medical need. BridgeBio Pharma aims to change this with their investigational…
Cooking and eating together is a great way to build closer bonds and spend time with those we love. In Malaysia, it's a large part of the culture, as there…
Aurinia Pharmaceuticals Inc. plans to release the updated results from their continuation study of AURORA 2. This clinical trial is evaluating LUPKYNIS, also known as voclosporin, as a treatment for…
Early diagnosis is critical in many rare diseases, as earlier intervention leads to better treatment outcomes. Now, diagnosis is becoming easier and quicker through the use of artificial intelligence (AI).…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
