Results 101 to 110 of about 406 (138)

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

Exploring the boundaries of Niemann-Pick disease type A/B: a report of a case and review of literature. [PDF]

Mol Cell Pediatr
El-Mezayen M, Tawfik AM, Hadhoud AM, Gerges VM, Afify MH.   +4 more
europepmc   +1 more source

Advanced strategies for detecting acid sphingomyelinase deficiency type B with attenuated phenotypes. [PDF]

Orphanet J Rare Dis
Villeneuve T, Jamme T, Schwob R, Levade T, Prévot G.   +4 more
europepmc   +1 more source

Re-anchoring the Value of Innovative Therapies in NICE Decision Making When Comparators are Cost Ineffective: A Case Study of Late-Onset Pompe Disease. [PDF]

Pharmacoeconomics
Walton M, Deng NJ, Corbett M, Umemneku-Chikere C, Nevitt SJ, Fulbright H, Tan CY, Lachmann R, Churchill R, Hodgson R.   +9 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