GPRC5D-Targeting Approach Positions Company to Expand Post-BCMA Treatment Options As reported on Fierce BioTech, Bristol Myers Squibb announced positive results from its pivotal Quintessential trial
Editor’s Note: We’re honored to share the following article from our friends at the Courageous Parents Network. To see this article in its original format,
Editor’s Note: Patient Worthy is honored to share this article from Rareatives, originally written by our friend Jessica Lynn. To see the article in its
Editor’s Note: Patient Worthy is honored to share this story from our friends at Elephants & Tea, originally written by Bill Phillips, a colorectal cancer
A single genetic change can have dramatically different effects from one person to the next. That is the reality for people living with PTEN hamartoma
As reported on QZ, the U.S. Food and Drug Administration has approved a new indication for Eli Lilly’s tirzepatide (Mounjaro), allowing the therapy to be
As reported on MedCity News, the U.S. Food and Drug Administration has approved Takeda Pharmaceutical’s Mimrylo (rusfertide) for the treatment of erythrocytosis in adults with
As reported on Drugs, BioNTech has decided to halt development of its Phase 2 BNT122-01 study investigating autogene cevumeran (BNT122/RO7198457) in patients with resected colorectal
As reported on PharmaBiz, BeOne Medicines has reported positive results from the second interim analysis of the phase 3 HERIZON-GEA-01 trial, further supporting the use
At 22, a young person’s world should be filled with possibilities—a career to build, places to travel, friendships to make, and a future to imagine.
As reported on Pharmaceutical Technology, the US Food and Drug Administration (FDA) has granted approval to PharmaEssentia’s Besremi (ropeginterferon alfa-2b-njft) for the treatment of adults
In a recent press release, Novartis has reported positive topline results from its Phase III REMODEL-1 and REMODEL-2 trials, showing that the investigational oral Bruton’s
As reported on Business Wire, the European Commission has approved trastuzumab deruxtecan (Enhertu) in combination with pertuzumab for the first-line treatment of adults with unresectable
As reported in the European Medical Journal, Novartis has paused eight clinical studies evaluating its experimental CAR-T cell therapy, known as rap-cell, after three trial
As reported in the European Medical Journal, new research suggests that cherry angiomas occur more frequently and at an earlier age in people with neurofibromatosis
In a recent press release, BioCryst Pharmaceuticals has shared that it received approval from Japan’s Ministry of Health, Labour and Welfare for the use of
As reported on Inside Precision Medicine, a new, non-invasive test may help doctors detect oral cancer quickly and reduce the need for painful scalpel biopsies.
Have you ever looked in the mirror and wondered, “Who am I now? Why did this diagnosis happen to me? When will this be over?
wefunder.com
Advancing precision therapies for rare neurodevelopmental diseases. Mahzi Therapeutics is testing the waters on Wefunder. Reserve your interest and foll...
Next Generation of Cystinosis #saturday #Sept19thIn-person registration and virtual attendance options!
#Cystinosis #LysosomalDisease #Research #ReimagineRare #SocialHealth
[ID: announcement from Engage Health Inc. declaring: we're presenting at WORLDFair 2026 (hosted by Dr Jeanine Jarnes University of Minnesota)
Learn about
Social and Psychological Adult
Health in Cystinosis
Featuring our speaker
Austin Letcher, MS
Director, Program & Operations, Engage Health
Along with our partner
Author Rebekah Palmer
Co-Founder & Advisor
Next Generation of Cystinosis
Attending? Reach out to Anne Melchior to connect!
[email protected] or
651-994-0510
September 18, 2026
9:30am - 4pm CST
Minnesota Landscape Arboretum
Chaska, MN
worldfair.health/worldfair]
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