Pregnant women now often receive promotional material from private cord blood banks urging them to save and store their babies' cord blood.

The media recently reported that the FDA has threatened to withdraw Synthroid from the market, alarming many physicians and patients.

Recombinant human α-L-iduronidase (laronidase; Aldurazyme BioMarin/Genzyme), an orphan drug, has been approved by the FDA for enzyme replacement therapy in mucopolysaccharidosis (MPS) type I, a lysosomal storage disorder caused by deficiency of the enzyme. This review describes the disease, treatment, adverse effects and cost.

The FDA has approved the ophthalmic solutions riboflavin 5'-phosphate in 20% dextran (Photrexa Viscous – Avedro) and riboflavin 5'-phosphate alone (Photrexa) for use with ultraviolet A (UVA) light (KXL System) in the corneal collagen cross-linking treatment of two corneal disorders: progressive keratoconus and corneal ectasia following refractive surgery. It is the first corneal collagen cross-linking (CXL) treatment to be approved by the FDA.

hydrochloride (Ticlid - Syntex), a platelet aggregation inhibitor that has been available in Europe for more than a decade, was recently approved by the U.S. Food and Drug Administration to decrease the risk of thrombotic stroke in men and women who have had premonitory signs of stroke or have had a completed thrombotic stroke. The manufacturer is promoting the drug for patients intolerant to aspirin or with conditions for which aspirin has not been approved (previous thrombotic stroke; transient ischemic attacks in women). The drug has also been used for prevention of myocardial ...

The FDA has approved Opill (Perrigo), a progestin-only oral contraceptive that contains norgestrel, for sale over the counter (OTC). Opill is the first oral contraceptive to be approved in the US for sale without a prescription. Approval of OTC Opill is intended to increase access to effective contraception and reduce the rate of unintended pregnancies.

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 Aurlumyn (Eicos Sciences), an IV formulation of the prostacyclin analog iloprost, to reduce the risk of digit amputation in adults with severe frostbite. Iloprost is the first drug to be approved by the FDA for treatment of severe frostbite; it has been used off-label for this indication in the EU and elsewhere for decades. In the US, iloprost is also available as an inhaled formulation (Ventavis) for treatment of pulmonary arterial hypertension.

Interim results of a double-blind, placebo-controlled trial suggest that off-label use of the anticholinergic drug oxybutynin may reduce the frequency and severity of hot flashes in women with breast cancer. Extended-release oral oxybutynin (Ditropan XL, and generics) has been shown to reduce the frequency and severity of hot flashes in healthy menopausal women.

Many women of child-bearing age now take folic acid supplements of 400 ╡g per day to prevent neural tube defects in their offspring (Medical Letter 1998; 40:75). An FDA advisory committee has recommended development of a combination tablet containing an oral contraceptive and 400 ╡g of folic acid. A recent editorial in the New England Journal of Medicine suggested that folic acid doses should be higher (NJ Wald, N Engl J Med 2004; 350:101).