Results 151 to 160 of about 110,217 (215)

Towards a sustainable rare disease and orphan drug ecosystem in Saudi Arabia: policy insights from a multi-stakeholder workshop. [PDF]

Front Pharmacol
Abozaid GM, Al-Omar HA, Alrabiah A, AlMuaither A, McKnight AJ.   +4 more
europepmc   +1 more source

Evolving Research and Development Landscape for Rare Diseases: Growing Concerns Over Orphan Drug Lag in Japan. [PDF]

Clin Pharmacol Ther
Enya K, Kageyama I, Kobayashi Y, Lim Y, Sengoku S, Kodama K.   +5 more
europepmc   +1 more source

Successes and pitfalls in orphan drug development for sickle cell disease. [PDF]

Blood Adv
Costa E, Isgrò A, de Montalembert M, Leufkens HGM, Ware RE, De Franceschi L.   +5 more
europepmc   +1 more source

Adverse Reactions to the Orphan Drug Cerliponase Alfa in the Treatment of Neurolipofuscinosis Type 2 (CLN2). [PDF]

Pharmaceuticals (Basel)
Ammendolia I, Sframeli M, Esposito E, Cardia L, Noto A, Currò M, Calapai G, De Pasquale M, Mannucci C, Calapai F.   +9 more
europepmc   +1 more source

Drug Price and Health Policy Knowledge Influence Prescription Behavior in Orphan Diseases: Pheochromocytoma and Paraganglioma As Prototypes of Orphan Drug Econometrics. [PDF]

Cureus
Le DQ, Burton BN, Tejeda CJ, Hong JC, Lee JS, Kamdar N.   +5 more
europepmc   +1 more source

Navigating health policies and programs in India: exploring opportunities to improve rare disease management and orphan drug research. [PDF]

Orphanet J Rare Dis
Mishra S, Bhat D, Venkatesh MP.
europepmc   +1 more source

Advancing orphan drug development for rare diseases. [PDF]

Clin Exp Pediatr
Ko JM.
europepmc   +1 more source

Generic orphan drug substitution: a critical analysis of global practices and Saudi Arabia's perspective. [PDF]

Front Pharmacol
Alakeel YS, Rampakakis E, AlRumaih A, AlRuwaisan R, Abushal M, AlDalaan AM, Idrees MM, Alanazi ZD, AlKoait H, Muaadi A, AlAfra MAM, AlShaya SA, AlHomida S.   +12 more
europepmc   +1 more source

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

Pediatric Orphan Drug Indications: 2010-2018. [PDF]

Pediatrics, 2020
Kimmel L, Conti RM, Volerman A, Chua KP.
europepmc   +1 more source