Recent research identifies novel genetic causes for rare movement and neurodegenerative disorders, including CD99L2 variants in spastic ataxia, PPP2R5C as an Alzheimer's biomarker, and toxic polyglycine proteins in GGC repeat expansion diseases.
Global orphan drug sales are forecast to reach $409 billion by 2032, representing one-fifth of prescription drug sales, with Johnson & Johnson and Argenx leading the market amid regulatory uncertainty.
Weill Cornell Medicine has launched an enterprise-wide AI to Advance Medicine initiative featuring a bimonthly lecture series, grant program, and new website to support safe and effective artificial intelligence use across research, education, and clinical care.
Roche deployed 2,176 new NVIDIA Blackwell GPUs, bringing its total above 3,500 chips across on-premise and cloud infrastructure. The expansion follows Eli Lilly's 1,016-GPU supercomputer launch and Recursion's 504-GPU system unveiled in 2024.
Lupin has signed a license and supply agreement with Spektus Pharma to commercialize DeslaFlex, a novel antidepressant for Major Depressive Disorder, in Canada, strengthening its CNS portfolio.
Merck will present late-breaking clinical data from its cardio-pulmonary pipeline at ACC.26, including positive Phase 3 results for enlicitide, an investigational oral PCSK9 inhibitor, and Phase 2 data for WINREVAIR in heart failure patients.
Diagnosing rare diseases can take 3 to 15 years, while about 95% lack approved treatments. Families are advocating for regulatory changes to speed access to therapies as new digital tools aim to improve clinical trials.
A redesigned CD40 agonist antibody achieved complete remission in two of 12 patients with metastatic cancer in early trials, while separate research identified a method to prevent deadly heart complications from immune checkpoint inhibitors.
New NICE guidance recommends offering SGLT-2 inhibitors earlier in type 2 diabetes treatment, potentially preventing 17,000 deaths over three years while saving the NHS £560 million through generic medicines.
ACT Capital Management invested $7.25 million in Viking Therapeutics as the company advances its obesity drug candidate VK2735 into Phase 3 trials. India's drug regulator warned pharmaceutical companies against direct or surrogate advertising of weight-loss medicines, while South Korean drugmakers target year-end commercialization of domestically developed obesity treatments.
At least seven people in California, Florida, and Texas have been sickened by E. coli linked to cheddar cheese made from raw milk by California-based Raw Farm. The company has declined to voluntarily recall its products.
Johnson & Johnson settled two cosmetic talc cases in March 2026, one before jury selection in Florida and another after opening statements in Washington, as the company faces multiple trials scheduled for April.
Bio-Techne reported quarterly results ahead of earnings and revenue forecasts, attracting fresh institutional buying. Despite the earnings beat, shares declined 15-20% over 30 days and posted losses over one year.
Recent studies demonstrate high response rates for antibody-drug conjugate in rare blood cancer, improved outcomes with metastasis-directed radiation in prostate cancer, and a novel drug combination strategy for ovarian cancer resistance.
PrimeC, an oral combination therapy, showed safety and signals of clinical benefit in a phase 2b trial, while PLL001, a gut-targeting therapy, demonstrated positive safety and tolerability in a phase I/II study for amyotrophic lateral sclerosis.
Northwestern Medicine scientists discovered that N-acetyl-L-leucine (NALL), an FDA-approved compound, promotes neuroprotective effects in experimental Parkinson's disease models by targeting multiple molecular pathways simultaneously.
HCW Biologics regained compliance with Nasdaq listing rules and completed a follow-on offering priced at $0.6055 per unit to raise approximately $1.5 million for clinical development of immunotherapies targeting chronic inflammation.
Two phase 3 trials demonstrate significant improvements in skin clearance and disease severity for children and adolescents with moderate-to-severe atopic dermatitis treated with targeted biologics.
Cogent Biosciences has submitted a New Drug Application for bezuclastinib to treat systemic mastocytosis caused by the KIT D816V mutation, with FDA review underway and strong analyst support.
Eli Lilly announced positive Phase 3 results for EBGLYSS in pediatric atopic dermatitis, orforglipron in diabetes showing superiority over Rybelsus, and Retevmo in early-stage lung cancer, expanding potential indications across its portfolio.