Results 61 to 70 of about 392 (96)

Evolution of Movement Disorders in Patients With CLN2-Batten Disease Treated With Enzyme Replacement Therapy. [PDF]

Neurology
Spaull R, Soo AK, Batzios S, Footitt E, Whiteley R, Mink JW, Carr L, Gissen P, Kurian MA.   +8 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

Role of Biomarkers in Diagnosing Disease, Assessing the Severity and Progression of Disease, and Evaluating the Efficacy of Therapies. [PDF]

J Inherit Metab Dis
Schiffmann R.
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