Results 21 to 30 of about 526 (116)

2019 FDA TIDES (Peptides and Oligonucleotides) Harvest

Pharmaceuticals, 2020
2019 has been an excellent year in terms of peptides and oligonucleotides (TIDES) approved by the FDA. Despite the drop in the number of total drugs approved by the FDA in 2019 in comparison with 2018 (48 vs.
Danah Al Shaer, Othman Al Musaimi, Fernando Albericio, Beatriz G. de la Torre   +3 more
doaj   +1 more source

Real-world assessment of the patient profile, clinical characteristics, treatment patterns, and outcomes associated with erythropoietic and X-linked protoporphyria. [PDF]

J Dermatol
Abstract Erythropoietic protoporphyria (EPP) and X‐linked protoporphyria (XLP) are rare genetic disorders. There are limited data regarding how these disorders are managed in real‐world settings. The aim of this study was to document the characteristics and treatment patterns among patients diagnosed with EPP or XLP in general real‐world settings in ...
Silver SM, Houghton K, Hitchens A, Ansquer VD, Ciepielewska M.   +4 more
europepmc   +2 more sources

Afamelanotide in Protoporphyria and Other Skin Diseases: A Review [PDF]

Postepy Dermatologii I Alergologii
Adriana Polańska, Petra Staubach-Renz, Aleksandra Dańczak-Pazdrowska   +2 more
exaly   +2 more sources

Increased phototoxic burn tolerance time and quality of life in patients with erythropoietic protoporphyria treated with afamelanotide – a three years observational study

Orphanet Journal of Rare Diseases, 2020
Background Erythropoietic protoporphyria (EPP) is an ultra-rare genetic disorder (prevalence 1:150`000) characterized by instant painful phototoxic burn reactions in skin exposed to visible light.
Jasmin Barman-Aksözen, Michèle Nydegger, Xiaoye Schneider-Yin, Anna-Elisabeth Minder   +3 more
doaj   +1 more source

Erythropoietic protoporphyria:time to prodrome, the warning signal to exit sun exposure without pain—a patient-reported outcome efficacy measure

, 2021
Purpose: Patients with erythropoietic protoporphyria (EPP), a severe painful photodermatosis, experience prodromal sensations when exposed to sunlight, which are the “warning signals” to exit the sun, as prolonged exposure causes an excruciatingly ...
Naik, H., Wheeden, K., Balwani, M., Overbey, J. R., Van Broekhoven, E. J.E., Desnick, R. J., Wagenmakers, M. A.E.M., Wilson, J. H.P., Wensink, D., Langendonk, J. G.; id_orcid   +9 more
core   +2 more sources

An Update on Vitiligo Target Therapy: A Semi-Structured Systematic Review [PDF]

, 2023
Background: There is growing evidence to suggest a significant role for the adaptive immune system in disease progression and relapse. Therapies targeted the inflammatory responses of epidermal and immune system cells have been demonstrated effect in the
Morrissey, Hana, Al Abadie, Miriam, Ball, Patrick Anthony, Al Abadie, Mohammed, Tukmatchy, Hussain   +4 more
core   +4 more sources

Quality of life correlations in patients with Erythropoietic Protoporphyria and X-Linked Protoporphyria on Afamelanotide: A retrospective cohort study - Supplemental Figure 1

, 2022
Investigating the impact of therapeutic response to afamelanotide on quality of life in patients with erythropoietic protoporphyria and x-linked protoporphyria.THIS DATASET IS ARCHIVED AT DANS/EASY, BUT NOT ACCESSIBLE HERE.
Ceresnie, M (via Mendeley Data), Ceresnie, Marissa   +1 more
core   +1 more source

The Impact of Minimal Sunlight Exposure on Bone Health: Insights From a Cohort Study in Erythropoietic Protoporphyria. [PDF]

J Clin Endocrinol Metab
Context: Erythropoietic protoporphyria (EPP) is a rare inherited metabolic disease, causing lifelong painful phototoxic reactions, minimal sunlight exposure, and vitamin D deficiency.
Kluijver LG, Wagenmakers MAEM, Wilson JHP, Langendonk JG.   +3 more
europepmc   +2 more sources

Patient empowerment and access to medicines: Insights from a scientist-patient suffering from erythropoietic protoporphyria

Medicine Access @ Point of Care, 2019
Patient representation during the evaluation of medicines by key decision makers such as regulatory agencies, Health Technology Assessment bodies, and healthcare payers is increasingly considered to add value to the appraisals and empowers patients ...
Jasmin Barman-Aksözen
doaj   +1 more source

Patient-recorded outcome to assess therapeutic efficacy in protoporphyria-induced dermal phototoxicity: a proposal

Health and Quality of Life Outcomes, 2010
Background Protoporphyria (PP) resulting from two rare, inherited diseases of heme biosynthesis leads to dermal phototoxicity by accumulation of the heme precursor protoporphyrin IX.
Minder Christoph E, Schneider-Yin Xiaoye, Minder Elisabeth I   +2 more
doaj   +1 more source