Suchergebnisse - "Rare disease"

PubPharm (589)

1

GOunder/Desmoid Tumor Research Foundation DEsmoid Symptom/Impact Scale ($ GODDESS^{©} $): psychometric properties and clinically meaningful thresholds as assessed in the Phase 3 DeFi randomized controlled clinical trial

enthalten in: Quality of life research | 2023

von Gounder, M. | Atkinson, T. | Bell, T. | +5

2

A Qualitative Research Study of Experiences and Perceptions of People Living with Insulin Resistance Syndrome or Lipoatrophic Diabetes in Japan

enthalten in: Diabetes therapy | 2023

von Yagi, N. | Toda, A. | Mitani, K. | +2

3

Cardiovascular risk in rare diseases: a prognostic stratification model in a cohort of sarcoidosis patients

enthalten in: Internal and emergency medicine | 2023

von Rizzi, L. | Coppola, C. | Cocco, V. | +2

4

Impact of SARS-CoV-2 infection in patients with hereditary hemorrhagic telangiectasia: epidemiological and clinical data from the comprehensive Italian retrospective multicenter study

enthalten in: Internal and emergency medicine | 2023

von Suppressa, P. | Maiorano, E. | Gaetani, E. | +15

5

The GAIN Registry — a New Prospective Study for Patients with Multi-organ Autoimmunity and Autoinflammation

enthalten in: Journal of clinical immunology | 2023

von Staus, P. | Rusch, S. | El-Helou, S. | +23

6

Grundlagen der Diagnostik primärer Immunstörungen („inborn errors of immunity“)

enthalten in: Zeitschrift für Rheumatologie | 2023

von Geier, C. | Voll, R. | Warnatz, K.

7

Decision Paralysis: Recognition and Patient-Centered Discourse

enthalten in: Dermatology and therapy | 2023

von Schreidah, C. | Fahmy, L. | Lapolla, B. | +1

8

Parents’ Experience of Administering Vosoritide: A Daily Injectable for Children with Achondroplasia

enthalten in: Advances in therapy | 2023

von NiMhurchadha, S. | Butler, K. | Argent, R. | +4

9

Patient Centricity : Design and Conduct of Clinical Trials in Orphan Diseases: Third of Three Sets of Expanded Proceedings from the 2020 ISCTM Autumn Conference on Pediatric Drug Development

enthalten in: Innovations in clinical neuroscience | 2023

von Busner, J. | Pandina, G. | Day, S. | +4

10

Australian children living with rare diseases: health service use and barriers to accessing care

enthalten in: World journal of pediatrics | 2023

von Teutsch, S. | Zurynski, Y. | Eslick, G. | +6

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