Rare Diseases

Rare Diseases Perspective

  • 5 Things to Know About CIDP While a rare, often perplexing disease, chronic inflammatory demyelinating polyneuropathy is a treatable condition.
  • Stop Incentivizing Drug Companies for More Rare Disease Drugs   Time to revise the Orphan Drug Act, which gives extra payment to companies that develop drugs for rare diseases. The Act has fueled a price explosion and possibly hindered research into other drugs.

Rare Diseases News & Journal Articles

  • FDA OKs Nintedanib (Ofev) for Rare Lung Disease Nintedanib capsules are approved for slowing the rate of decline in pulmonary function in adults with systemic sclerosis-associated interstitial lung disease.
  • Novel Therapeutic Approaches to Xeroderma Pigmentosum This review explores novel therapeutic approaches to xeroderma pigmentosum, focusing on targeting the molecular abnormalities that occur secondary to defects in nucleotide excision repair.

Medscape Editorial Collaborations