Results 81 to 90 of about 69,398 (132)

Persistent Treatment Effect of Cerliponase Alfa in Children with CLN2 Disease: A 3 Year Update from an Ongoing Multicenter Extension Study

, 2019
Nicola Specchio, Peter Slasor, Temitayo Ajayi, David Jacoby, Paul Gissen, Angela Schulz, Heather Cahan, Emily de los Reyes   +7 more
core   +1 more source

Access to Orphan Medicinal Products in Bulgaria: An Analysis of the Positive Drug List and Individual Access Schemes. [PDF]

Healthcare (Basel)
Stefanov R, Raycheva R, Kostadinov K, Stefanov G, Zdravkova-Aneva I, Dzhambazova E, Iskrov G.   +6 more
europepmc   +1 more source

External controls for rare disease drug development: Lessons for emerging and advanced therapeutic modalities. [PDF]

Mol Ther Adv
Hughes SH, Beretich LA, Fuller M, Goodspeed K, Penn M, Valentino LA, McCombs C.   +6 more
europepmc   +1 more source

Exploring the potential of natural history studies for rare neurological diseases. [PDF]

Ann Med Surg (Lond)
Muili AO, Gomez A, Sulaiman AO, Mutata S, Akhunji MT, Teemer A, Shahzad A, Kuol PP.   +7 more
europepmc   +1 more source

The Italian Assessment System for Innovative Medicines: An 8-Year Retrospective Analysis of Outcomes, Criteria, Timelines and Funding Dynamics. [PDF]

Appl Health Econ Health Policy
Martellone L, Servidio C, Vaccaro M, Malandrini K, Coluccia A, Oliva A, Mastroianni CM, Polito G.   +7 more
europepmc   +1 more source

Bridging the blood-brain barrier: strategies to improve delivery of biologics to tumors in the brain. [PDF]

Fluids Barriers CNS
Gampa G, Vadlakonda R, Stefanich E, Kamath AV, Sadekar S, Shivva V.   +5 more
europepmc   +1 more source

Strategy for reproductive and developmental toxicity (DART) studies for marketing applications in pharmaceutical development. [PDF]

Curr Res Toxicol
Kagawa Y, Osawa T, Koyama N, Tanaharu T, Kigami D.   +4 more
europepmc   +1 more source

Cerliponase alfa for neuronal ceroid lipofuscinosis type 2 disease.

Australian prescriber
openaire   +1 more source

Clinical pharmacology considerations for first-in-human clinical trials for enzyme replacement therapy. [PDF]

J Inherit Metab Dis
Stern S, Wang J, Li RJ, Hon YY, Weis SL, Wang YC, Schuck R, Pacanowski M.   +7 more
europepmc   +1 more source

A systematic review of real-world evidence (RWE) supportive of new drug and biologic license application approvals in rare diseases. [PDF]

Orphanet J Rare Dis
Vaghela S, Tanni KA, Banerjee G, Sikirica V.   +3 more
europepmc   +1 more source