As reported on drugs.com, REGENXBIO has disclosed that the U.S. Food and Drug Administration has halted clinical testing of its investigational gene therapies RGX‑111 and RGX‑121, both being developed for…
The European Medicines Agency (EMA) has issued a positive opinion recommending that retifanlimab (Zynyz) be approved for an expanded indication to treat adults with advanced squamous cell carcinoma of the…
As reported on Drugs.com, REGENXBIO has announced that the U.S. Food and Drug Administration (FDA) has halted clinical testing of its investigational gene therapies RGX‑111 and RGX‑121, two programs targeting…
As reported on Science Daily, a breast cancer vaccine tested more than 20 years ago is drawing renewed scientific interest after researchers discovered that every participant in the original trial…
As reported on BioSpace, Moderna has entered a commercialization partnership with Italy‑based Recordati to support the development of mRNA‑3927, an investigational mRNA therapy for propionic acidemia (PA). The agreement, announced…
A growing body of research shows that sleep quality, dietary sodium, and hypertension are more closely linked than once believed — and two major healthcare organizations are joining forces to…
As reported by the European Pharmacy Review, a combination approach using Johnson & Johnson’s small‑molecule therapy Caplyta (lumateperone) alongside standard antidepressants continues to show promising results for adults with major…
A newly published Phase 1/2 study in The New England Journal of Medicine, and reported on by the Manila Times, highlights promising early results for tividenofusp alfa (DNL310), Denali Therapeutics’ investigational…
As reported on Healio, glucagon-like peptide-1 (GLP-1)–based therapies are rapidly reshaping the landscape of diabetes and chronic kidney disease (CKD) management. Originally developed for glycemic control, these agents now play…
Immunoglobulin A nephropathy (IgAN) remains one of the most common primary glomerular diseases worldwide, yet effective treatments are limited. A new study, recently published in Frontiers, offers fresh evidence that…
As reported by Clinical Trials Arena, Axsome Therapeutics has begun dosing patients in its FORWARD Phase III clinical trial, a pivotal study designed to evaluate the investigational therapy AXS‑14 (esreboxetine)…
As reported on PM Live, a new clinical trial hub focused on epidermolysis bullosa (EB) has been established at University Medical Center Groningen (UMCG) in the Netherlands through a collaboration…
As discussed in a recent interview between Krystal Jacques and Prof. Brian Bigger, a novel stem cell–based gene therapy is showing early promise as a potential one-time, lifelong treatment for…
As reported on MedPage Today, an oral targeted therapy may offer a safe and effective alternative to injectable treatment for children with achondroplasia, according to new phase II data published…
Editor's Note: Patient Worthy is honored to share this post from Martha Harlam, Director of the Solution Project. Are you a student or researcher passionate about the future of neurology?…
As reported on Business Wire, ImmunityBio has released new clinical findings suggesting that its interleukin‑15 superagonist, ANKTIVA® (nogapendekin alfa inbakicept), may help restore immune function and improve survival outcomes in…
The U.S. Food and Drug Administration (FDA) has approved Yartemlea (narsoplimab‑wuug), marking the first authorized therapy for hematopoietic stem cell transplant‑associated thrombotic microangiopathy (TA‑TMA) in adults and children aged 2…
As reported by Technology, a major evidence synthesis published in The BMJ has delivered the clearest picture to date of which therapies offer the strongest short‑term benefits for people with…
As reported on PharmaBiz, AstraZeneca’s phase III LATIFY trial has fallen short of its primary objective, with the combination of the ATR inhibitor ceralasertib and the PD‑L1 inhibitor Imfinzi (durvalumab)…
As reported by The Pharma Letter, the UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted a new indication for argenx’s Vyvgart (efgartigimod alfa), expanding the therapy’s use to…
As reported on Forbes, a class of drugs first introduced to combat HIV is now demonstrating unexpected promise for patients with rare genetic and mitochondrial disorders. Once designed to block…
As reported on FierceBioTech, Biohaven closed out the holiday period with disappointing news: its investigational Kv7.2/7.3 potassium‑channel modulator, BHV‑7000, did not demonstrate efficacy in a phase 2 clinical trial for…
As reported by NewsWise, new clinical study led by investigators at the University of North Carolina at Chapel Hill is offering rare hope for families affected by Hunter syndrome, a…
As reported by Forbes, the U.S. Food and Drug Administration has granted approval to Flow Neuroscience for the first prescription brain stimulation device designed for home use in treating major…
As reported on Science Daily, a recent clinical study suggests that a traditional Chinese herbal remedy may offer a unique advantage in treating major depressive disorder (MDD) while paving the…
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