Top 5 Articles of the Week

Every Friday, we like to list the 5 most popular stories of the week.  This week, news from the FDA dominated with 2 approvals given by the agency as well as a breakthrough designation. Also hot this week was gene therapy and better screening of AATD patients. For these and other news stories, go to

FDA Approves Higher Dose of Orfadin for HT-1

The FDA also approved a higher dose of Orfadin (nitisinone; 20 mg) for the treatment of Hereditary Tyrosinemia type-1 (HT-1).

Breakthrough Therapy Designation Given  to Eosinophilic Esophagitis Drug

Patients with eosinophilic esophagitis may have persistent or relapsing symptoms related to esophageal dysfunction, which include dysphagia (difficulty swallowing) and food impaction.

FDA Approves At-home Treatment for AAT Deficiency

The FDA has approved an expanded label for Glassia [Alpha-1 Proteinase Inhibitor (Human)], so that adult patients with emphysema due to severe alpha-1 antitrypsin (AAT) deficiency can now self-infuse the orphan drug at home.

New Data From uniQure's Gene Therapy for Hemophilia B is Impressive

As of 6 months following gene therapy, all patients saw improvements in their disease phenotype and achieved sustained increases in FIX activity.

Should Emphysemia Patients be Screened for Alpha-1-antitrypsin Deficiency?

Results of a recent study published in Orphanet Journal of Rare Diseases suggest that targeted screening programs can successfully increase the rates of diagnosis for the genetic disease alpha-1-antitrypsin deficiency (AATD).

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