Rare Diseases

Rare Diseases Perspective

  • CDC's Emergency Drugs for US Clinicians and Hospitals   Suspect a case of botulism or malaria? You'll soon find that your local pharmacy doesn't stock antitoxin or artesunate. You can get these lifesaving drugs quickly from CDC. Here's how.
  • Newborn Screening for Rare Diseases   A parent's story with two different outcomes illustrates why newborn screening is so important in the early diagnosis and treatment of rare disorders.

Rare Diseases News & Journal Articles

  • Case Studies in Cystinuria What do these case studies tell us about the diagnosis and treatment of cystinuria?
  • British Drugs Body to Challenge New Cost Rules in Court Britain's pharmaceuticals trade body plans to go to court to challenge new cost rules for medicines for very rare diseases, describing the measures on Monday as 'inappropriate and unworkable'.

Medscape Editorial Collaborations