Patients with Duchenne muscular dystrophy (DMD) who received delandistrogene moxeparvovec (Elevidys; Sarepta Therapeutics) showed long-term disease stabilization or slowed disease progression at 3 and 5 years of follow-up in data presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific […]
Delandistrogene Moxeparvovec Shows Long-Term Impacts at 3 and 5 Years in DMD Read More »
The FDA approved oral iptacopan (Fabhalta; Novartis) to reduce proteinuria in adults with C3 glomerulopathy (C3G), according to a press release from Novartis.1 Iptacopan is now the first and only FDA-approved treatment for C3G, an ultrarare, progressive kidney disease. Iptacopan
FDA Approves Oral Iptacopan for C3 Glomerulopathy Read More »
Twenty years after the initial discussions of value-based insurance design (VBID), which at its core aims to foster the use of high-value health care services while minimizing the utilization of low-value services, there is still plenty of work to be
After 20 Years of VBID Discussion, Low-Hanging Fruit Remain Unpicked Read More »
Orca-T, an investigational allogeneic T-cell immunotherapy, improved chronic graft-vs-host disease (cGVHD)–free survival compared with allogeneic stem cell transplant (allo-HSCT) in patients with hematologic malignancies in the phase 3 Precision-T trial (NCT05316701), according to a press release from Orca Bio.1 Allo-HSCT
Orca-T Immunotherapy Improves cGVHD-Free Survival in ALL, AML, MDS Read More »
Multiparametric quantitative MRI (qMRI) effectively differentiated between Becker muscular dystrophy (BMD) and Duchenne muscular dystrophy (DMD) in ambulatory children in a study published in the Journal of Magnetic Resonance Imaging.1 The findings suggest multiparametric qMRI could help differentiate between DMD
MRI Could Aid in Early Differential Diagnosis of DMD, BMD Read More »
Low-density granulocyte (LDG) and normal-density neutrophil (NDN) subset changes identified in a recent study suggest that emergency hemopoiesis may be a first mechanism to maintain peripheral blood counts, but also a proinflammatory microenvironment, in patients with myelodysplastic syndromes (MDS), acute
Neutrophil Subset Changes Give Insight Into First Mechanisms of MDS, AML Read More »
Global initiatives to harmonize regulations for cell and gene therapies (CGTs)—including evidence standards, reporting requirements, and regulatory processes—could facilitate more timely access to innovative therapies, according to a research letter published in JAMA Internal Medicine. The study found that just
Uniform Global Standards, Processes Could Accelerate CGT Approvals, Access Read More »
The FDA approved revakinagene taroretcel-lwey (Encelto; Neurotech Pharmaceuticals) for the treatment of macular telangiectasia type 2 (MacTel), a neurodegenerative disease of the retina causing progressive and irreversible vision loss, Neurotech announced in a press release.1 Revakinagene taroretcel-lwey is the first
FDA Approves Revakinagene Taroretcel for Macular Telangiectasia Type 2 Read More »
If Congress eliminates enhanced premium tax credits (PTCs), the net cost of marketplace premiums would rise, and many Americans would become ineligible for tax credits to offset the cost of health insurance premiums, a report published by The Commonwealth Fund
Eliminating Enhanced PTCs Would Have Cascading Economic Impacts, Report Estimates Read More »
The FDA today approved tislelizumab (Tevimbra; BeIGene) plus chemotherapy for the first line treatment of patients with unresectable or metastatic esophageal squamous cell carcinoma (ESCC) whose tumors express PD-L1, according to a press release from BeiGene.1 Results from the phase
First-Line Tislelizumab Plus Chemotherapy Approved in ESCC Expressing PD-L1 Read More »
