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FDA Approves New Powder Formulation of Trofinetide (Daybue) for Rett Syndrome

20 minutes ago

The approval of a dye- and preservative-free powder formulation of trofinetide offers a new option for adult and pediatric patients with Rett syndrome.

Apremilast Effective Treatment for Moderate to Severe Genital Psoriasis at 32 Weeks

29 minutes ago

The phase 3 DISCREET study explored the safety and efficacy of apremilast in treating patients with moderate to severe genital psoriasis over 32 weeks.

Getting Through the Door: How COPD, Lung Cancer Screening Remains Minimal

41 minutes ago

2025 data show the US is still failing to adequately identify and initiate care for deadly lung disease. Experts discuss what it will take to turn the tide on this public health issue.

Efficacy and Safety of Zasocitinib in Psoriasis and the Ideal Candidates for TYK2 Inhibitors

1 hour ago

What are your impressions of the Phase 2b data of zasocitinib in psoriasis, particularly in terms of Psoriasis Area and Severity Index (PASI) responses, dose-dependent effects, and tolerability? How do you see these results influencing your real-world patient selection?

Evaluating Efficacy and Safety of Vutrisiran in ATTR-CM from the HELIOS-B Trial

1 hour ago

The panel reviews the HELIOS-B Phase 3 trial evaluating vutrisiran, a transthyretin silencer, in patients with ATTR-CM. Participants were randomized to receive vutrisiran or placebo, with approximately 40% already taking tafamidis at trial entry, particularly in the United States. The study was designed with predefined primary and secondary endpoints, assessing outcomes both in the overall population and in those not on baseline tafamidis. The trial demonstrated statistically significant results across all major endpoints, including reductions in all-cause mortality and cardiovascular hospitalizations, with notable benefit observed both in monotherapy and combination-therapy subgroups. The panel highlights the magnitude of treatment effect and notes that these findings supported FDA approval of vutrisiran for ATTR-CM. They also emphasize its favorable safety profile, explaining that common adverse events—such as cardiac failure and arrhythmias—reflect the underlying disease rather than the drug itself. Overall, the experts describe vutrisiran as a meaningful and well-tolerated therapeutic advancement for patients with ATTR-CM.

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