A major breakthrough for the 300 Irish patients living with butterfly skin. For the first time in Ireland, patients battling epidermolysis bullosa, a rare and
Editor’s Note: Patient Worthy is honored to share this story submitted to us by Regina Portnoy, a clinical researcher with over 20 years of experience.
Editor’s Note: Patient Worthy is honored to share this patient story from our friends at Elephants & Tea. To see the story in its original
As shared on drugs.com, Vascarta Inc. has reported encouraging results from a Phase 1b clinical study evaluating its investigational topical/transdermal curcumin gel, VAS-101, for the
Editor’s Note: Patient Worthy is honored to share this article from our friends at the Brighter Hope Foundation. To see the article in its original
Labcorp announced the nationwide availability of Agilent Technologies’ PD-L1 IHC 22C3 pharmDx, the only FDA-approved companion diagnostic designed to identify patients with platinum-resistant ovarian cancer
As reported on drugs.com, Revolution Medicines has reported practice-changing efficacy results from a pivotal Phase 3 trial evaluating daraxonrasib in patients with metastatic pancreatic ductal
This, year the PCBers will meet in Indianapolis, Indiana. They will be celebrating 30 years as a community, as well as celebrating a much brighter
Johnson & Johnson announced that the U.S. Food and Drug Administration (FDA) has granted Priority Review to IMAAVY® (nipocalimab-aahu), marking a significant milestone as the
As reported on Business Wire, Enveda, a clinical-stage biotechnology company focused on discovering novel small-molecule medicines, has reported encouraging results from a Phase 1b study
A recent article from Inside Precision Medicine states: For decades, systemic lupus erythematosus (SLE) has resisted tidy solutions, a shapeshifting autoimmune disease that crosses into
AstraZeneca has announced positive results from the pivotal phase III MIRANDA trial of tozorakimab, a groundbreaking first-in-class monoclonal antibody designed to treat Chronic Obstructive Pulmonary
On January 20, 2014, at 54 years old, I experienced what I casually called an “anxiety numbness event.” My left thumb and index finger went
The US Food and Drug Administration (FDA) has approved Idvynso, a groundbreaking two-drug single-tablet regimen combining 100 mg doravirine and 0.25 mg islatravir, for treating
My story began very suddenly on May 4th, 2025, when I was home from graduate school visiting my family. It had been a great day,
Glioblastoma multiforme (GBM) is the most common malignant brain tumor in adults and remains one of the most difficult cancers to treat. Characterized by aggressive
WebMD published a story about a young woman, Maddie, who had spent nine years in wheelchair confinement resulting from a rare metabolic disease related to
Health Canada has officially approved Cysklar® (cysteamine ophthalmic solution) 0.44%, marking a significant breakthrough for Canadians living with cystinosis. According to BusinessWire.com, the approval enables
Cystinosis Research NetworkCystinosis is a rare, genetic, metabolic, lysosomal storage disease that causes an abnormal accumulation of the amino acid cystine in various organs and tissues of the body.
For more info, visit our website (linked in comments)
#cystinosis #RareEdu #CystinosisAware
Jordan's Guardian Angels Northern Ireland Rare Disease PartnershipIrelandRareDiseasePartnership
The Life Raft Group #GistStay up to date on the latest #GISTresearch presented at ASCO 2026! Join Dr. Hirotaka Miyashita of UC San Diego Health on June 17 as he reviews key abstracts and presentations relevant to the #GISTcommunity.
Register today: bit.ly/UpdatesASCO2026
#GIST #ASCO26 #CancerResearch
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