As reported on PharmaBiz, the US Food and Drug Administration (FDA) has approved Orca Bio’s Tregzi, a precision-engineered cell therapy designed for use in matched-donor
As reported on FiercePharma, the U.S. Food and Drug Administration (FDA) has approved an expanded indication for Vertex Pharmaceuticals’ gene-editing therapy Casgevy (exagamglogene autotemcel), allowing
As reported on PharmaBiz, Ascletis Pharma has announced the submission of two Investigational New Drug (IND) applications to the US Food and Drug Administration (FDA)
As reported on Pharmaceutical Technology, the European Commission has approved Novartis’ gene replacement therapy Itvisma (onasemnogene abeparvovec) for the treatment of children aged two years
As reported on BioSpace, BridgeBio Pharma has reported new findings suggesting that its transthyretin (TTR) stabilizer, Attruby (acoramidis), may help preserve kidney function in patients
As reported on Healio, new phase 3 data suggest that sonelokimab may offer durable disease control for patients with moderate to severe hidradenitis suppurativa (HS),
New research from the 2026 American Society of Clinical Oncology (ASCO) annual meeting, and presented on Cure Today, highlights an encouraging new treatment on the
Editor’s Note: Patient Worthy is honored to share this article from our friend Abigail Johnston, originally published on her blog No Half Measures. To see
In recent reports on MSN and AdventLS, it was shared that, as a result of the pioneering efforts of Dr. Carla Nester and researchers at
Our friends and contributors at the TREND Community have provided us with another enlightening article, this time about high-quality human-generated data becoming a scarce and
What Happened? The healthcare company Grifols announced in a recent press release that it has enrolled and dosed the very first patient in a new
Editor’s Note: Patient Worthy is proud and honored to share this article, provided by our friends at The Assistance Fund. Every year, thousands of families
As reported on BioSpace, Praxis Precision Medicines has received a three-month extension to the U.S. Food and Drug Administration’s review of relutrigine, its investigational therapy
Early Clinical Results Highlight Potential New Treatment Strategy As reported on STAT News, a novel investigational drug combination developed through a collaboration between Tango Therapeutics
As reported on Becker’s Hospital Review, the FDA has approved risankizumab (Skyrizi) for use in children and adolescents aged 6 years and older with moderate-to-severe
According to STAT News, a major international clinical trial is about to begin in the Democratic Republic of the Congo (DRC) as health authorities respond
As reported on PharmaBiz, the U.S. Food and Drug Administration (FDA) has approved Lumvoa (veligrotug‑vvze), a new therapy developed by Viridian Therapeutics, for the treatment
As reported by PharmaBiz, Eli Lilly and Company has announced positive findings from its Phase 3 BRUIN CLL-322 trial evaluating pirtobrutinib (Jaypirca), a next-generation non-covalent
#rarecancer @occularmelanoma #melanoma🧩 What if every cancer is rare... when it's your cancer?
This thought-provoking article explores a new way of thinking about ocular melanoma and why every rare cancer deserves awareness, research, and support.
Read more: acureinsight.org/is-every-cancer-a-rare-cancer-a-new-way-of-thinking-about-ocular-melanoma/
💙 Together, we can make a difference.
#go to your conference LGS Foundation
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Charcot-Marie-Tooth Association @VandaPharmaceuticals #cmt2sCMTA-STAR Alliance Partner Vanda Pharmaceuticals announced the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to VCA-894A, its investigational therapy for Charcot-Marie-Tooth disease type 2S (CMT2S). Learn more: cmtausa.org/simply-cmt/vanda-pharmaceuticals-fda-rare-pediatric-disease-designation-cmt2s/
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