Results 161 to 170 of about 88,273 (287)

Thrombospondin‐4 is upregulated in abdominal aortic aneurysm: A vasoprotective response with potential therapeutic relevance

British Journal of Pharmacology, EarlyView.
TSP4 is upregulated in human and experimental AAA. TSP4 knockdown accelerated AAA formation and exacerbated both vascular inflammation and remodelling, emphasizing that TSP4 upregulation may represent a protective response against AAA. Our findings suggest that preserving TSP4 could be a useful strategy in the clinical management of AAA.
Laia Blanco‐Casoliva, Lidia Puertas‐Umbert, Judith Alonso, Rafael Almendra‐Pegueros, Saray Varona, Mercedes Camacho, Gemma Arderiu, Lluís Asmarats, Marta Alegret, Jose Martinez‐Gonzalez, Cristina Rodriguez   +10 more
wiley   +1 more source

Expectations and Concerns of Adoptive Families of Children With Fetal Alcohol Spectrum Disorder

Child &Family Social Work, EarlyView.
ABSTRACT Fetal alcohol spectrum disorder (FASD) is considered a condition that persists throughout life in people who have been exposed to alcohol during the gestation period. During the different stages of their lives, individuals with FASD face different challenges and adversities that—without the right resources and strategies—can contribute to ...
Dídac Gómez‐Santabrígida, Carme Montserrat, Marta Garcia‐Molsosa, Gala Lima‐López   +3 more
wiley   +1 more source

Informing Dose for Pediatric Rare Diseases-A Survey of Recent Orphan Drugs Approvals. [PDF]

Clin Transl Sci
Fletcher EP, Kim C, Mei H, AbuAsal B, Samuels S, Madabushi R, Ramamoorthy A.   +6 more
europepmc   +1 more source

Cutaneous and non‐cutaneous diseases due to Mycoplasma pneumoniae in children

JDDG: Journal der Deutschen Dermatologischen Gesellschaft, EarlyView.
Summary Mycoplasma pneumoniae (MP) is a common pathogen responsible for diverse infections in children and adolescents, primarily affecting the respiratory tract. Besides causing atypical pneumonia, MP can also lead to extrapulmonary manifestations, including mucocutaneous, hematological, neurological, cardiac, and gastrointestinal symptoms.
Hanna Lindemann, Maximilian David Mauritz, Victoria Rehmann, Michael Paulussen, Jorge Frank, Malik Aydin   +5 more
wiley   +1 more source

Lifecycle management of orphan drugs approved in Japan. [PDF]

Orphanet J Rare Dis, 2022
Seki K, Suzuki H, Abe S, Saotome C.
europepmc   +1 more source

Clinical and genetic characterization of intellectual disability

Developmental Medicine &Child Neurology, EarlyView.
This study examines the etiological factors and comorbidities in a large cohort of Finnish patients with intellectual disability. Genetic causes—including chromosomal abnormalities and pathogenic gene variants—were more frequently identified in individuals with moderate to profound intellectual disability.
Aarni Venetvaara, Minna Kraatari‐Tiri, Jussi‐Pekka Tolonen, Marko Merikukka, IDGEN Study Group, Hannaleena Kokkonen, Teija Paakkola, Susanna Varis, Antti Dahl, Jarmo Körkkö, Jonna Komulainen‐Ebrahim, Jukka Moilanen, Johanna Uusimaa, Outi Kuismin, Elisa Rahikkala   +14 more
wiley   +1 more source

Navigating market access after conditional reimbursement: a communication roadmap for disinvesting orphan drugs. [PDF]

Int J Technol Assess Health Care
Abdallah K, Blonda A, Godman B, Huys I, Claes K, Denier Y, Simoens S.   +6 more
europepmc   +1 more source

Top Selling (2026) Small Molecule Orphan Drugs: A Journey into Their Chemistry. [PDF]

Int J Mol Sci, 2023
Benedetto Tiz D, Bagnoli L, Rosati O, Marini F, Sancineto L, Santi C.   +5 more
europepmc   +1 more source

Metabolic liver disease: A summary of major guidelines and identifying opportunities to improve future guidelines

Diabetes, Obesity and Metabolism, EarlyView.
Abstract The clinical management of metabolic dysfunction‐associated steatotic liver disease (MASLD) and metabolic dysfunction‐associated steatohepatitis (MASH) is undergoing rapid evolution, driven by advances in noninvasive diagnostics and the recent approval of liver‐directed therapies.
Fernando Bril
wiley   +1 more source

Societal participation in the development of orphan drugs: a systematic review. [PDF]

Front Med (Lausanne)
Akodad S, De Smedt D, Stevens H.
europepmc   +1 more source