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May 16, 2026
A case report details a 63-year-old woman with insulin autoimmune syndrome and Hashimoto thyroiditis, presenting with hypoglycemia as low as 27 mg/dL. Elevated insulin and autoantibody titers confirmed the diagnosis. Prednisolone therapy rapidly improved glycemic stability, and the patient remained euglycemic after tapering.
Jul 10, 2026
Phase III data show deramiocel slowed upper limb decline by 54% in Duchenne muscular dystrophy. The FDA is re-evaluating the BLA with HOPE-3 results, with a submission of the clinical study report expected in February 2026.
Mar 29, 2026
Priovant Therapeutics' brepocitinib demonstrated significant efficacy in the Phase 3 VALOR trial for dermatomyositis, meeting its primary endpoint with a 15.3-point greater improvement in Total Improvement Score versus placebo at Week 52. The treatment showed meaningful corticosteroid tapering and improvements across all nine key secondary endpoints, with benefits sustained through one year. Additional analyses revealed rapid itch reduction and improved skin-related quality of life in patients.
Mar 09, 2026
Five intra-articular gene therapy platforms for knee osteoarthritis are in human trials in the US, while India prepares regulatory guidelines for mRNA, gene and cell therapies to accelerate innovation.
Feb 26, 2026
The FDA cleared NexEos Bio's IND application to proceed with a Phase II/III trial of NTX-1024 ophthalmic solution for vernal keratoconjunctivitis, following positive results from a 15-patient study in Mexico.
Feb 21, 2026
Long-term data from the QUASAR extension study show TREMFYA® maintained clinical remission in over 80% of ulcerative colitis patients at Week 140, with more than half achieving endoscopic remission and no new safety concerns observed.
Feb 14, 2026
Ocular Therapeutix announced positive topline results from SOL-1, its Phase 3 superiority trial of AXPAXLI for wet age-related macular degeneration, meeting primary and durability endpoints with high statistical significance.
Feb 13, 2026
PTC Therapeutics has withdrawn its New Drug Application for Translarna (ataluren) for Duchenne muscular dystrophy after the FDA indicated the data were unlikely to meet approval thresholds, ending a pursuit spanning more than a decade.
Feb 13, 2026
PTC Therapeutics has withdrawn its New Drug Application resubmission for ataluren (Translarna) for nonsense mutation Duchenne muscular dystrophy after the FDA indicated the submission lacked substantial evidence of effectiveness.
