Orphan Drugs (a Drug Or Biological Product For The Diagnosis Or Treatment Of A Rare Disease), A Disease That Effects >< Than 200,000 People

Question 0

a1-Proteinase inhibitor (Prolastin)

Answer 0

Replacement therapy for congenital a1-proteinase (a1-antitrypsin) deficiency

Question 1

Acetylcysteine

Answer 1

Intravenous admin. For moderate to severe acetaminophene overdose

Question 2

Beractant (Survanta)

Answer 2

Prevention or treatment of RDS in newborns

Question 3

Cystic fibrosis transmembrane conductance regulator

Answer 3

Treatment of cystic fibrosis

Question 4

Dornase alfa (pulmozyme)

Answer 4

Treatment of cystic fibrosis: reduction of mucus viscosity and increase in airway secretion clearance

Question 5

Nitric oxide gas (Inomax)

Answer 5

Treatment of persistent pulmonary hypertension of newborns, or of acute respiratory distress in adults

Question 6

Tobramycin solution for inhalation (TOBI)

Answer 6

Treatment of pseudomonas aeruginosa in cystic fibrosis or bronchiectasis

Question 7

Pentamidine isethionate

Answer 7

Prevent pneumocystis carinii (jiroveci) pneumonia in high risk patients

Question 8

Respiratory syncytial virus immune globulin intravenous (human)

Answer 8

Prophylaxis of (respiratory syncytial virus) lower respiratory tract infections in infants and young children at risk of RSV.