As reported by Manila Times, Ocular Therapeutix has released additional one‑year results from its Phase 3 SOL‑1 trial evaluating AXPAXLI (formerly OTX‑TKI) for the treatment
As reported on BioPharmaDive, several biopharmaceutical companies reported notable clinical and regulatory developments this week, with investor response reflecting growing confidence in both late‑stage pipelines
Researchers have demonstrated substantial therapeutic benefits for brepocitinib, an oral tyrosine kinase 2/Janus kinase 1 inhibitor, in adults with dermatomyositis, according to findings from a
Akebia Therapeutics has initiated clinical development of a new investigational therapy aimed at preventing or treating acute kidney injury (AKI) associated with cardiac surgery. The
Triple negative breast cancer, the most aggressive form of breast cancer, is under attack. Adelaide University researchers, led by Dr. Theresa Hinkey, report that unlike
CatalYm has initiated patient enrollment in the GDFATHER-HCC-01 trial, a Phase 2b study investigating visugromab as part of a combinatorial treatment strategy for patients with
Phase 2 data suggest meaningful skin improvement and an acceptable safety profile As reported on Healio, litifilimab, an investigational therapy developed by Biogen, demonstrated encouraging
As reported on Inside Precision Medicine, Researchers at Stanford have created a new urine test that helps doctors understand how well bladder cancer treatments are
Chugai Pharmaceutical Co., Ltd. announced that Roche has made the strategic decision to discontinue clinical development of GYM329 (emugrobart), an investigational anti-latent myostatin antibody, for
As reported on PharmaBiz, Amgen has announced encouraging topline results from a phase 3 clinical trial evaluating a subcutaneous formulation of Tepezza (teprotumumab-trbw) for adults
Most of us have heard of the fight-or-flight stress response. It becomes activated when we believe there is a chance we can outfight or outrun
Telomir Pharmaceuticals has taken a significant step forward in the development of a novel therapeutic agent for one of oncology’s most challenging cancers. As reported
I have battled several autoimmune and neurological conditions that are triggered by food and other factors for over 50 years. It took most of my
The traditional model of HIV care has long centered on one fundamental requirement: patients must take medication every single day, without fail. Yet this model
As reported by GlobeNewsWire, results from the Phase 3 VALOR trial evaluating brepocitinib in adults with dermatomyositis have been published in the New England Journal
Editor’s Note: Patient Worthy is pleased to share the final part in a series of excerpts by Dana Langston. Chapter 36: Holding the Line Knowing
For patients living with primary biliary cholangitis (PBC), a rare autoimmune liver disease, an often-overlooked symptom has plagued their existence, an intense, maddening internal itch
As reported on PharmaBiz, a novel treatment strategy combining dual immunotherapy, targeted therapy, and transarterial chemoembolization (TACE) has demonstrated meaningful clinical benefit for patients with
Understanding how SMA is tested is a key step toward earlier diagnosis and faster access to care. From genetic blood testing to newborn screening, modern tools are helping identify spinal muscular atrophy sooner and more accurately than ever before. #spinalmuscularatrophy #genetictesting #PatientWorthy
International Foundation for CDKL5 Research-IFCRHave you registered for the CDKL5 Family Conference yet? You won’t want to miss this.👇
We’re hosting a powerful, honest session on gene therapy - starting with real talk about where the science stands, what risks exist, and what expectations are realistic.
Then we’ll move into a community risk/benefit workshop where every voice matters. Whether you’re eager to join a trial, unsure, or not interested at all, we need your perspective to understand the full picture of our community’s hopes and concerns.
Your voice shapes the future - We hope to see you there!! 💜✨
#Conference2026 #GeneTherapy #EducationalFamilyConference #RareDiseaseCommunity #CDKL5 #IFCRCommunity
United Mitochondrial Disease FoundationLooking for connection and support? Join patients and families from the mito community at one of UMDF’s virtual Support Meetings. Visit our events calendar for login details and come be part of the conversation. umdf.org/events-calendar/
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