Results 61 to 70 of about 5,265,871 (311)

Badian, Sam (Box 90, Folder 5)

, 1976
Series VIII: Audiocassettes features informal oral histories about the Hebrew Orphan Asylum and its alumni.Digital recordingDigital finding ...
Hebrew Orphan Asylum of New York
core  

The Multiple Criteria Qualitative Value‐Based Pricing Framework “MARIE” for Novel Cell and Gene Therapy

Clinical Pharmacology &Therapeutics, EarlyView.
When setting the price of cell and gene therapies in Japan, the factors considered include the manufacturing and operating costs of the new drug or a comparator, leading to discrepancies between price and values, in particular those specific to cell and gene therapies.
Akina Takami, Ataru Igarashi
wiley   +1 more source

Unmet Needs in Darier’s Disease from a Patient’s Perspective: Lessons Learnt from the German Registry

Acta Dermato-Venereologica
The MDHHgermany registry was initiated to characterize the “real-life” situation of affected individuals with Darier’s disease (DD; Morbus Darier, MD) and Hailey-Hailey disease (HH), including their treatment and healthcare.
Danielle Rogner, Laura Heimerl, Tilo Biedermann, Elke Sattler, Alexander Zink   +4 more
doaj   +1 more source

Berman, Hilda (Box 90, Folder 7)

, 1986
Series VIII: Audiocassettes features informal oral histories about the Hebrew Orphan Asylum and its alumni.Digital recordingDigital finding ...
Hebrew Orphan Asylum of New York
core  

Long‐Acting Therapeutics in Pediatric Health: Bridging Innovation and Access

Clinical Pharmacology &Therapeutics, EarlyView.
Children have distinct therapeutic needs arising from age‐dependent physiology, disease epidemiology, formulation requirements, dosing considerations, and safety vulnerabilities. Despite substantial reductions in childhood mortality over the past three decades, nearly 5 million children under five died in 2023, with infectious diseases continuing to ...
Prajith Venkatasubramanian, Moherndran Archary, Daniel B. Hawcutt, Rachel Daley, Edmund Capparelli, Adeniyi Olagunju   +5 more
wiley   +1 more source

Targeting Aromatase and 5-α-Reductase to Limit Dysfunctional Adipogenesis in Multiple Symmetric Lipomatosis

Biomedicines
Background: Multiple symmetric lipomatosis (MSL) is an orphan disease characterized by pathological expansion of lipomatous tissue (LT) in the subcutaneous adipose tissue (SAT) areas, causing severe deformities and compression of vital organs.
Gabriella Milan, Chiara Compagnin, Isabel Zucal, Giuseppe Perale, Silvia Bettini, Anna Pilatone, Marco De Monti, Yves Harder, Corrado Parodi, Daniel Schmauss, Vincenzo Vindigni, Franco Bassetto, Oliver Felthaus, Dmytro Oliinyk, Lukas Prantl, Luca Busetto   +15 more
doaj   +1 more source

Orphan Disease: Infantile Systemic Hyalinosis

Zdorovʹe Rebenka, 2015
Infantile systemic hyalinosis — one of the forms of systemic hyaline fibromatosis — is a rare, fatal progressive disease with autosomal recessive inheritance, which is characterized by the accumulation of hyaline substance in different tissues of the ...
Ye.V. Ponochevnaia, Ye.N. Okhotnikova, O.F. Zarudniaia, V.T. Leush, Ya.I. Doronina, Ye.I. Usova, I.V. Hedeon   +6 more
doaj   +1 more source

Adults with LCH – orphans with an orphan disease [PDF]

Clinical Medicine, 2006
Tatevossian R, Nanduri V, Salotti J, Sargent C, Norton A, Wells A, Pritchard J   +6 more
openaire   +2 more sources

Kaplan, Ted (Box 90, Folder 23)

, 1976
Series VIII: Audiocassettes features informal oral histories about the Hebrew Orphan Asylum and its alumni.Digital recordingDigital finding ...
Hebrew Orphan Asylum of New York
core  

Transcriptomic Profiling of SLC and ABC Transporters in the Human Term Placenta

Clinical Pharmacology &Therapeutics, EarlyView.
Solute carriers (SLC) and ATP‐binding cassette (ABC) transporters are essential for placental solute exchange and fetal protection, yet their transcriptomic profiles in the human placenta remain poorly characterized. Although fetal sex influences placental development and function, its impact on transporter expression is unclear.
Elijah Marsh Jung, Nicholas Ieronimakis, Bothaina Mousa, Emily D. Sheikh, Sarah E. Ligon, Joshua W. Monson, Jennifer R. Damicis, Hillary J. Kinsman, Lydia E. Bettridge, Samuel L.M. Arnold, Edward J. Kelly, Jashvant D. Unadkat, Joanne Wang   +12 more
wiley   +1 more source