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 the treatment to be used…
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) for experimental obesity treatments designed…
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 and older, adolescents, and adults…
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 with transthyretin amyloidosis cardiomyopathy (ATTR-CM),…
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), with more than two-thirds of…
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 horizon for advanced non-small cell…
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 the article in its original…
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 the Children’s Hospital of Iowa…
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 valuable asset as AI companies…
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 Phase 3 clinical trial called…
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 receive a rare disease diagnosis.…
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 for rare genetic forms of…
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 and Revolution Medicines has demonstrated…
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 plaque psoriasis who are appropriate…
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 to a rapidly escalating outbreak…
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 of thyroid eye disease (TED),…
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 Bruton tyrosine kinase (BTK) inhibitor,…
As reported on PharmaTimes, the United Arab Emirates has taken a significant step toward preventive neurology with the initiation of its first clinical trial guided by population-scale genomics. Spearheaded by…
As reported by Yahoo Finance, the U.S. Food and Drug Administration (FDA) has accepted for review two applications from Sandoz for generic versions of tirzepatide, a GLP-1–based therapy currently marketed…
As reported on FiercePharma, the U.S. Food and Drug Administration (FDA) has selected seven pharmaceutical and biotechnology companies to participate in its newly established PreCheck Pilot Program, an initiative aimed…
As reported on Healio, the U.S. Food and Drug Administration has broadened the indication for roflumilast cream 0.3% (Zoryve, Arcutis), allowing its use in children with plaque psoriasis as young…
Editor's Note: Patient Worthy is honored to share this story from our friends at Elephants & Tea, originally written by Amy Hoffmann — a survivor of triple-negative breast cancer. To…
One of the hardest things about living with dystonia is trying to explain what it feels like on the inside. People often see (not always for some) the muscle contractions, the pulling, the tension…
Editor's Note: Patient Worthy is honored to share this article, provided by our friends at the TREND Community. To see the article in its original format, please click here. Healthcare…
Editor's Note: This article was originally published by Patient Worthy on 6/7/24, and is being republished due to our ongoing efforts to raise awareness of Hidradenitis Suppurativa (HS). The importance…
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