Results 71 to 80 of about 172,212 (109)

Efficacy of different treatment strategies in patients with mucopolysaccharidosis: a systematic review and network meta-analysis of randomized controlled trials. [PDF]

Orphanet J Rare Dis
Huang L, Wu J, Tang B, Wu J, Wei F, Li HQ, Li L, Wang X, Wang B, Wu W, Hong X.   +10 more
europepmc   +1 more source

Alfa N°56 Plante Margarine

ALFA N°56 PLANTE MARGARINE Alfa N°56 Plante Margarine ( -

core  

Novel transferrin receptor-mediated enzyme replacement therapy efficiently treats myogenic and neurogenic aspects of Pompe disease in mice. [PDF]

Mol Ther Methods Clin Dev
George K, Riley R, Zhou S, Allen E, Smith L, Kistanova E, Kinton S, Guo L, Bangari D, Ismail A, Thummapudi J, Boumezbeur F, Selingue E, Tamarelle D, Capdevila C, Do TM, Lesuisse D, Leksa N, Sardi P, van der Flier A.   +19 more
europepmc   +1 more source

A pH-sensitive binding modality allows successful transferrin receptor-mediated transcytosis of a bivalent antibody across brain barriers. [PDF]

MAbs
Kuhn P, Petralla S, Dabbagh F, Pegoretti V, Muranyi W, Ishikawa H, Schroten H, Fischer R, Frenzel A, Schirrmann T, Rehm M, Schwerk C, Fricker G, Kontermann R, Fullstone G.   +14 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

Therapeutic oligonucleotides as genetic medicines: The current state and future perspective. [PDF]

Cell Rep Med
Godinho BMDC, Ferguson CM, Aartsma-Rus A.   +2 more
europepmc   +1 more source

Clinical data required for the approval of pediatric pharmaceuticals in Japan. [PDF]

BMC Pediatr
Haigo H, Matsuda K, Shikano M.
europepmc   +1 more source

Targeting Neurological Aspects of Mucopolysaccharidosis Type II: Enzyme Replacement Therapy and Beyond. [PDF]

BioDrugs
Zanetti A, Tomanin R.
europepmc   +1 more source

Rapid genotyping of inversion variants in Mucopolysaccharidosis type II using long-range PCR: A case report. [PDF]

Mol Genet Metab Rep
Hattori Y, Kido J, Sugawara K, Sawada T, Matsumoto S, Nakamura K.   +5 more
europepmc   +1 more source

Breaking Biological Barriers: Engineering Nanocarriers for Efficient Drug and Gene Delivery through Nano-Bio Interfaces and Biophysical Design. [PDF]

Appl Phys Rev
Deng Z, Dunn A, Sun DQ, Shi D.
europepmc   +1 more source