Results 131 to 140 of about 109,656 (184)

The Orphan Drug Act at 40: Legislative Triumph and the Challenges of Success. [PDF]

Milbank Q
Saltonstall P, Ross H, Kim PT.
europepmc   +1 more source

FDA Approval of Orphan Drug Indications for Pediatric Patients, 2011-2023.

JAMA Pediatr
Kakkilaya A, Shahzad M, Bourgeois FT.
europepmc   +1 more source

Orphan Drug [PDF]

, 2020
openaire   +1 more source

Predictors of orphan drug approval

Orphanet Journal of Rare Diseases, 2010
Heemstra Harald
doaj   +1 more source

Patents, Innovation and Access to New Pharmaceuticals [PDF]

Henry Grabowski
core  

Pharmacogenetic Interventions, Orphan Groups, and Distributive Justice: The Role of Cost-Benefit Analysis [PDF]

, 2002
Rai, Arti K.
core   +1 more source

Real-World Safety Data of the Orphan Drug Onasemnogene Abeparvovec (Zolgensma^®) for the SMA Rare Disease: A Pharmacovigilance Study Based on the EMA Adverse Event Reporting System. [PDF]

Pharmaceuticals (Basel)
Ruggiero R, Balzano N, Nicoletti MM, di Mauro G, Fraenza F, Campitiello MR, Rossi F, Capuano A.   +7 more
europepmc   +1 more source