Results 91 to 100 of about 676 (154)

Prenatal Delivery of Enzyme Replacement Therapy to Fetuses Affected by Early-Onset Lysosomal Storage Diseases. [PDF]

Am J Med Genet C Semin Med Genet
Borges B, Canepa E, Chang IJ, Herzeg A, Lianoglou B, Kishnani PS, Harmatz P, MacKenzie TC, Cohen JL.   +8 more
europepmc   +1 more source

Classification of Infiltrative Heart Diseases MORAL-STAGE System. [PDF]

Clin Med Insights Cardiol
Reznik EV, Nguyen TL, Semyachkina AN, Shkolnikova MA.   +3 more
europepmc   +1 more source

Clinical and molecular spectrum of mucopolysaccharidosis IVA in Iraqi children: Allele-specific genotype-phenotype trends and novel GALNS variants. [PDF]

Mol Genet Metab Rep
Wahhab SBA, Thejeal RF.
europepmc   +1 more source

Products with Black Box Warnings [PDF]

, 2006
Woelfel, Joseph A.
core   +1 more source

Quality in qualitative evidence: new best practice principles from NICE's real-world evidence framework. [PDF]

J Comp Eff Res
Gupta VA, Scott S, Tonkinson M, Jonsson P, Goodburn L, Duffield S.   +5 more
europepmc   +1 more source

How are patient inputs considered in HTA? A thematic document analysis of NICE ultra-rare disease appraisals. [PDF]

Eur J Health Econ
Gentilini A, Rana A.
europepmc   +1 more source

Drugs to Be Used With a Filter for Preparation and/or Administration-2024. [PDF]

Hosp Pharm
Ipema HJ, Denton C, Koppen L, Joseph H, Munir F, Arvis H, Coslow A, Rhodes A, Vazquez L.   +8 more
europepmc   +1 more source

A Rare Case of Morquio Syndrome in Palestine: Clinical, Radiological, and Genetic Insights. [PDF]

Cureus
Farah RE, Farah RE, Ayasa IW, Abuisneina S.   +3 more
europepmc   +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