Too Many Mutants with Multiple Mutations

Exon skipping for nonsense mutations in Duchenne muscular dystrophy: too many mutations, too few patients?

INTRODUCTION Duchenne muscular dystrophy (DMD), one of the most common and lethal genetic disorders, is caused by mutations of the dystrophin gene. Removal of an exon or of multiple exons using antisense molecules has been demonstrated to allow synthesis of truncated 'Becker muscular dystrophy-like' dystrophin. AREAS COVERED Approximately 15% of DMD cases are caused by a nonsense mutation. Al...

How many "me-too" drugs is too many?

ON AUGUST 3, 2009, THE US FOOD AND DRUG ADministration (FDA) approved pitavastatin (Livalo), making it the eighth statin approved for use in the United States and seventh currently available for sale. This approval comes almost a quarter century after that for the first member of the class, lovastatin, 8 years after generic lovastatin was approved and 4 years after 2 additional statins, pravast...

Therapeutic cytapheresis: too many platelets, too many white blood cells.

Myeloproliferative disorders may present with thrombocytosis and/or leukocytosis with symptoms ranging from minor to life-threatening. Therapeutic cytapheresis (TC), the removal of excess and/or abnormal cells such as platelets and white blood cells, can be used to rapidly reduce cells in the symptomatic patient. The patient's clinical status determines the need for TC as symptoms may not corre...

Too few physicians, or too many?

Rate Changes – Too many or too few ?