The adeno-associated virus (AAV) vector-based gene therapy delandistrogene moxeparvovec-rokl (Elevidys – Sarepta) received accelerated approval from the FDA in 2023 for treatment of ambulatory children 4-5 years old with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene. It has now received full approval for use in ambulatory patients ≥4 years old and accelerated approval for use in nonambulatory patients with DMD.

The amyloid beta-directed monoclonal antibody lecanemab-irmb (Leqembi – Eisai/Biogen) has now been approved by the FDA for once-monthly use for treatment of Alzheimer's disease (AD) in patients with mild cognitive impairment (MCI) or mild dementia. It was previously approved only for administration once every 2 weeks. Now, lecanemab can be given every 4 weeks after an 18-month initiation phase of biweekly administration.

The FDA has approved the nitrofuran antiprotozoal drug nifurtimox (Lampit – Bayer) for treatment of Chagas disease (American trypanosomiasis) in children who weigh ≥2.5 kg. Nifurtimox was previously available in the US only through a CDC-sponsored Investigational New Drug (IND) program.

In June 2021, the FDA approved the IV amyloid beta-directed monoclonal antibody aducanumab (Aduhelm) for treatment of Alzheimer’s disease. The approval did not restrict use of the drug to patients with mild cognitive impairment or mild dementia, which was the population enrolled in the clinical trials. Now, Biogen, with the permission of the FDA, has made an addition to the labeling of the drug that says: Treatment with Aduhelm should be initiated in patients with mild cognitive impairment or mild dementia stage of disease, the population in which treatment was initiated in clinical...

The FDA has removed the Risk Evaluation and Mitigation Strategy (REMS) requirement for the endothelin receptor antagonists ambrisentan (Letairis, and generics), bosentan (Tracleer, and generics), macitentan (Opsumit, and generics; Opsynvi), and aprocitentan (Tryvio), and the endothelin receptor antagonist and angiotensin II receptor antagonist sparsentan (Filspari) that was initially implemented because of a possible risk of embryofetal toxicity.

The FDA has approved the oral Janus kinase (JAK) inhibitor deuruxolitinib (Leqselvi – Sun) for treatment of severe alopecia areata in adults. Two other oral JAK inhibitors are approved for treatment of severe alopecia areata: baricitinib (Olumiant) is approved for use in adults, and ritlecitinib (Litfulo) is approved for use in patients ≥12 years old.

View the Comparison Table: Antiviral Drugs for Seasonal Influenza for 2025-2026

Anxiety disorders (generalized anxiety disorder, panic disorder, social anxiety disorder, and various phobias) are the most common form of psychiatric illness. They can be treated effectively with cognitive behavioral therapy (CBT) and/or pharmacotherapy.

Lyme disease in the US is caused by the spirochete Borrelia burgdorferi, which is transmitted to humans by Ixodes scapularis (blacklegged [deer] tick) and I. pacificus (western blacklegged tick). Most cases of Lyme disease occur in late spring and early summer in northeastern and mid-Atlantic states, the upper Midwest, and in northern California. B. mayonii, which is also transmitted by I. scapularis, has been shown to cause a similar illness in the upper Midwest.

Cognitive behavioral therapy for insomnia (CBT-I) is recommended for initial treatment of chronic insomnia. CBT-I includes stimulus control, sleep education and hygiene, sleep restriction, relaxation training, and cognitive therapy. When CBT-I alone is not effective, pharmacologic treatment should be added.