Results 61 to 70 of about 285 (98)

Acid sphingomyelinase deficiency with homozygous p.Arg610del genotype in an elderly patient: a rare case report. [PDF]

J Bras Pneumol
Bridi GDP, Kairalla RA, Sawamura MVY, Baldi BG.   +3 more
europepmc   +1 more source

Impact of Diffusing Lung Capacity for Carbon Monoxide on Mortality Risk in Patients with ASMD: Insights from a Post Hoc Analysis. [PDF]

Adv Ther
Wuyts WA, Bonella F, Scarpa M, Kapetanakis V, Shukla P, Fournier M, Gasparic M, Pulikottil-Jacob R.   +7 more
europepmc   +1 more source

More Than a Question of Correlation: Characterization of the Evidentiary Basis for Biomarker Surrogates Used in European Marketing Authorizations. [PDF]

Clin Pharmacol Ther
Grupstra RJ, Starokozhko V, Bakker E, Pasmooij AMG, Mol PGM.   +4 more
europepmc   +1 more source

Access to anticancer and orphan medicines through compassionate use programs and named patient basis in seven European countries. [PDF]

ESMO Open
Rosenberg N, Post HC, Schutte T, de Visser SJ, Bartelink I, Pasmooij AMG, van Laarhoven HWM, Hollak CEM.   +7 more
europepmc   +1 more source

A commentary on GST reforms and their effect on cancer care in India. [PDF]

Front Public Health
Sahni V, Shankar A.
europepmc   +1 more source

Unexplained splenomegaly as a diagnostic marker for a rare but severe disease with an innovative and highly effective new treatment option: A case report. [PDF]

Mol Genet Metab Rep
Van Baelen A, Verhulst S, Eyskens F.
europepmc   +1 more source

Acid sphingomyelinase deficiency and Gaucher disease: Underdiagnosed and often treatable causes of hepatomegaly, splenomegaly, and low HDL cholesterol in lean individuals. [PDF]

Hepatol Commun
Mistry PK, Cassiman D, Jones SA, Lachmann R, Lukina E, Prada CE, Wasserstein MP, Thurberg BL, Foster MC, Patel RM, Underhill LH, Peterschmitt MJ.   +11 more
europepmc   +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

Methodological challenges in Dutch HTA of non-oncological orphan drugs: a retrospective analysis and price comparison using different pricing models. [PDF]

Orphanet J Rare Dis
Walraven J, Kaveh M, Uyl-de Groot C.
europepmc   +1 more source