Delandistrogene moxeparvovec-rokl (Elevidys – Sarepta), an adeno-associated virus (AAV) vector-based gene therapy, has received accelerated approval from the FDA for treatment of ambulatory children 4-5 years old with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the dystrophin gene. It is the first gene therapy to be approved in the US for treatment of DMD. Accelerated approval was based on expression of microdystrophin in skeletal muscle, a surrogate endpoint.

The FDA has approved datopotamab deruxtecan (Datroway – Daiichi Sankyo/AstraZeneca), a trophoblast cell-surface antigen-2 (Trop-2)-directed antibody and topoisomerase inhibitor conjugate, for treatment of adults with unresectable or metastatic hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative breast cancer who received prior endocrine-based therapy and chemotherapy for unresectable or metastatic disease.

Tiopronin (Thiola - Mission), a thiol compound (mercaptopropionyl glycine) that solubilizes cystine by reacting with it to form a mixed disulfide, is now available in the USA for treatment of refractory cystinuria.

Eribulin mesylate (Halaven – Eisai) has been approved by the FDA for treatment of patients with metastatic breast cancer who have previously received at least 2 chemotherapy regimens for metastatic cancer. Prior therapy should have included an anthracycline and a taxane in either an adjuvant or metastatic setting. Other drugs used to treat anthracycline- and taxane-refractory metastatic breast cancer include capecitabine (Xeloda), gemcitabine (Gemzar, and others) and vinorelbine (Navelbine, and others).

Recent reports indicate that intramuscular (IM) injection of the nonsteroidal anti-inflammatory drug (NSAID) ketorolac (Toradol, and others), sometimes directly into injured muscles, has become a common practice in US locker rooms.

Full-page newspaper advertisements and a series of television commercials have urged patients with atrial fibrillation to "rethink warfarin" in favor of Eliquis (apixaban – Bristol-Myers Squibb). Apixaban is the latest of 3 new oral anticoagulants now competing with warfarin (Coumadin, and others) for the oral anticoagulant market.

Bivalirudin is being promoted as an intravenous (IV) anticoagulant that can be used instead of heparin in patients with unstable angina undergoing coronary angioplasty.

Alefacept (a le' fa sept; Amevive - Biogen) has been approved by the FDA for parenteral treatment of adults with moderate to severe chronic plaque psoriasis.

Levodopa combined with carbidopa (Sinemet, and others) is the most widely used treatment for patients with Parkinson's disease, but after 2 to 5 years most patients develop troublesome complications (Treatment Guidelines from The Medical Letter 2004; 2:41). The newest treatment for Parkinson's disease patients with end-of-dose "wearing-off" is Stalevo (Novartis), a combination of the catechol-O-methyltransferase (COMT) inhibitor entacapone (Comtan) with 3 different doses of levodopa/carbidopa. The rationale for Stalevo is that it permits some patients to take only one pill rather than...

Advertisements are appearing in medical journals for continuing medical education courses on catheter ablation of atrial fibrillation. Is a cure at hand for this common disorder?