Cogent Biosciences has announced new clinical trial results indicating that its drug BEZUCLASTINIB offers promising benefits for patients with indolent systemic mastocytosis (ISM), a rare and chronic disorder characterized by…
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…
Ready to read? Before you do, make sure to check out Parts 1 and 2 of Tracy's story. In Part 1, Tracy describes the symptoms that led her to pursue a diagnosis. Part 2…
Before you read on, make sure to check out Part 1 of Tracy's story. In Part 1, Tracy discusses the symptoms that led her to pursue a diagnosis and the multi-year diagnostic…
Ten years ago, Tracy Kyttle moved from New England to the DC Metro Area to pursue a career in education leadership. She loved the area’s duality: its proximity to nature…
Cogent Biosciences has just released promising initial data from their Phase 2 clinical trial called APEX. This trial is investigating bezuclastinib, a selective KIT D816V inhibitor, as a treatment for…
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…
According to a recent article in Biospace, patients with advanced systemic mastocytosis (SM) can turn to a newly FDA-approved treatment by Blueprint Medicines called Ayvakit (avapritinib). The drug is the…
As the pandemic closed bars and theaters and so many of our jobs became remote, much of the world became virtual. And as cocktail hour moved onto zoom and museums…
The first time I got stung by a wasp, my knee swelled up to nearly 4x its size. While my allergy is more frustrating than fatal, I know that I…
According to an article in Clinical Trials Arena, Blueprint Medicine has been steadily and speedily recruiting participants for its Phase II PIONEER trial, which examines Ayvakit as a treatment for…
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…
Blueprint Medicines Corporation has just announced that they have submitted a supplemental New Drug Application (NDA) to the FDA. This application is for a therapy called AYVAKIT, which was developed…
The 62nd American Society of Hematology (ASH) Annual Meeting and Exposition will be held virtually this year from December 5 through 8. The goal of the meeting is to provide…
In a recent press release, precision therapy company Blueprint Medicines Corporation announced positive results from two clinical trials: EXPLORER and PATHFINDER. The trials examined the safety, efficacy, and tolerability…
By Lauren Thayer from In The Cloud Copy Systemic mastocytosis (SM) is a complicated condition of the blood in which excessive numbers of mast cells (a type of white blood…
According to a story from mdmag.com, a phase 2 trial testing the drug avapritinib as a treatment for systemic mastocytosis is currently ongoing. The results so far suggest that the…
A PIONEER trial has been conducted in order to evaluate the effects of avapritinib, a drug meant to treat systematic mastocytosis. Researchers want to find a better treatment for…
According to a story from PR Newswire, the Blueprint Medicines Corporation recently announced that latest results from a Phase 1 clinical trial of avapritinib as a treatment for advanced systemic…
Promising data from the ongoing Phase I clinical trial Explorer has been announced by Blueprint Medicines Corporation. The trial is designed to evaluate the effects of the experimental drug avapritinib…
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.
