Guardant Health, Inc. (Nasdaq: GH), a leading precision oncology company, today announced that real-world evidence generated from Guardant’s InfinityAI contributed to the recent approval of ENHERTU® (trastuzumab deruxtecan), developed and commercialized by Daiichi Sankyo (TSE:4568) in Japan, for the treatment of patients with HER2-positive (HER2 [ERBB2] gene amplification or immunohistochemistry [IHC] 3+) advanced or recurrent solid cancers refractory or intolerant to standard treatments. This a

After Spinraza's FDA approval nearly a decade ago, Biogen's spinal muscular atrophy (SMA) drug captured rapid momentum and delivered blockbuster-level sales in just its second year on the market. While it's been a reliable revenue contributor ever since, rival meds from Roche and Novartis have taken away some of Spinraza's steam over the years. Now, Biogen is ready with its answer, as the FDA has approved the company's high-dose version of its SMA blockbuster. The new version features an "accel

Results of dose-ranging study suggest potential for best-in-class clinical profile across efficacy, safety and ease of use, including simplified dosing regimen with minimal to no titration necessary HRS-1893 treatment resulted in rapid and substantial reductions in left ventricular outflow tract gradient (Valsalva LVOT-G complete gradient response (<30 mmHg) of up to 86%) with minimal change in left ventricular ejection fraction (LVEF; range of decrease between 1.8% and 2.7%) Data highlight

March 30, 2026 -- Cure SMA, the leading nonprofit organization supporting those impacted by spinal muscular atrophy (SMA), celebrates that Biogen today announced FDA approval of the High Dose regimen of SPINRAZA™ (nusinersen) to treat people living with SMA. With broad label approval for all those living with SMA, this is an excellent result for the SMA community. SPINRAZA is an antisense oligonucleotide therapy that increases production of survival motor neuron (SMN) protein, essential for moto

Aurinia Pharmaceuticals Inc. (NASDAQ: AUPH), a biopharmaceutical company focused on delivering therapies to people living with autoimmune diseases with high unmet medical needs, announced today it has entered into a definitive merger agreement (the “Merger Agreement”) to acquire Kezar Life Sciences, Inc. (NASDAQ: KZR), a biotechnology company focusing on small-molecule therapeutics to treat unmet needs in autoimmunity and cancer, for $6.955 in cash per share of Kezar common stock, plus one non-t

March 30, 2026 -- Nanoscope Therapeutics, Inc., a biotechnology company committed to developing and commercializing novel, disease-agnostic therapies for patients with photoreceptor loss and vision impairment due to retinal degeneration, today announced that it has received Orphan Drug Designation in the Kingdom of Saudi Arabia for its lead therapy, MCO-010, for the treatment of patients with inherited retinal dystrophies (IRDs) having severe vision loss. The broad designation from Saudi regula

KalVista Pharmaceuticals, Inc. (Nasdaq: KALV) today announced new interim results from its KONFIDENT-KID clinical trial evaluating EKTERLY® (sebetralstat) for the on-demand treatment of hereditary angioedema (HAE) attacks in children ages 2-11 presented at the 2026 Global Angioedema Leadership Conference. KONFIDENT-KID is the largest pediatric trial ever conducted in HAE. It features a proprietary oral disintegrating tablet (ODT) formulation of sebetralstat in a population that currently relies

March 30, 2026 -- Sobi® (STO: SOBI) today announced that the European Medicines Agency (EMA) has validated an indication extension application for Tryngolza® (olezarsen) for the treatment of adult patients with severe hypertriglyceridemia (sHTG) ≥880 mg/dL (≥10 mmol/L). Patients with elevated triglyceride levels have substantially higher risks of all-cause mortality, atherosclerotic cardiovascular events, and acute pancreatitis[1]. The submission is supported by results from the pivotal Phase 3

Luxna Biotech Co., Ltd. and KH Neochem Co., Ltd.today announced the launch of a joint research initiative aimed at advancing next-generation drug discovery through the development of improved delivery technologies for nucleic acid therapeutics. Nucleic acid therapeutics are emerging as a promising modality for treating cancer and other challenging diseases. Unlike conventional small-molecule drugs and antibody therapeutics, they enable access to previously undruggable targets. However, instabi

Poster #76954 highlights QTORIN™ rapamycin’s single phase anhydrous gel formulation designed to optimize dermal bioavailability of rapamycin for mTOR-driven skin diseases while overcoming the crystallization, stability, and dermal penetration challenges posed by rapamycin Poster #76929 highlights a qualitative patient and caregiver interview study evaluating the burden of living with porokeratosis, a serious, rare genetic skin disease characterized by numerous pre-cancerous, pruritic lesions and