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Literature DB >> 30683214

Spotlight on rare diseases.

Abstract

Mesh：

Year: 2019 PMID： 30683214 DOI： 10.1016/S2213-8587(19)30006-3

Source DB: PubMed Journal: Lancet Diabetes Endocrinol ISSN： 2213-8587 Impact factor: 32.069

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Cited

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15 in total

1. The impact of the Orphan Drug Act on Food and Drug Administration-approved therapies for rare skin diseases and skin-related cancers.

Authors: Laura Karas; Christine Y Lu; Pankaj B Agrawal; Maryam M Asgari
Journal: J Am Acad Dermatol Date: 2019-05-16 Impact factor: 11.527

2. Pediatric Orphan Drug Indications: 2010-2018.

Authors: Lauren Kimmel; Rena M Conti; Anna Volerman; Kao-Ping Chua
Journal: Pediatrics Date: 2020-03-03 Impact factor: 7.124

Review 3. Artificial Intelligence in Medical Imaging and its Impact on the Rare Disease Community: Threats, Challenges and Opportunities.

Authors: Navid Hasani; Faraz Farhadi; Michael A Morris; Moozhan Nikpanah; Arman Rhamim; Yanji Xu; Anne Pariser; Michael T Collins; Ronald M Summers; Elizabeth Jones; Eliot Siegel; Babak Saboury
Journal: PET Clin Date: 2022-01

4. The EU Child Cohort Network's core data: establishing a set of findable, accessible, interoperable and re-usable (FAIR) variables.

Authors: Angela Pinot de Moira; Sido Haakma; Katrine Strandberg-Larsen; Esther van Enckevort; Marjolein Kooijman; Tim Cadman; Marloes Cardol; Eva Corpeleijn; Sarah Crozier; Liesbeth Duijts; Ahmed Elhakeem; Johan G Eriksson; Janine F Felix; Sílvia Fernández-Barrés; Rachel E Foong; Anne Forhan; Veit Grote; Kathrin Guerlich; Barbara Heude; Rae-Chi Huang; Marjo-Riitta Järvelin; Anne Cathrine Jørgensen; Tuija M Mikkola; Johanna L T Nader; Marie Pedersen; Maja Popovic; Nina Rautio; Lorenzo Richiardi; Justiina Ronkainen; Theano Roumeliotaki; Theodosia Salika; Sylvain Sebert; Johan L Vinther; Ellis Voerman; Martine Vrijheid; John Wright; Tiffany C Yang; Faryal Zariouh; Marie-Aline Charles; Hazel Inskip; Vincent W V Jaddoe; Morris A Swertz; Anne-Marie Nybo Andersen
Journal: Eur J Epidemiol Date: 2021-04-21 Impact factor: 12.434

Review 5. Perspectives of the European Society of Endocrinology (ESE) and the European Society of Paediatric Endocrinology (ESPE) on rare endocrine disease.

Authors: Martin Reincke; Anita Hokken-Koelega
Journal: Endocrine Date: 2021-03-19 Impact factor: 3.633

Spotlight on rare diseases.

1. The impact of the Orphan Drug Act on Food and Drug Administration-approved therapies for rare skin diseases and skin-related cancers.

2. Pediatric Orphan Drug Indications: 2010-2018.

Review 3. Artificial Intelligence in Medical Imaging and its Impact on the Rare Disease Community: Threats, Challenges and Opportunities.

4. The EU Child Cohort Network's core data: establishing a set of findable, accessible, interoperable and re-usable (FAIR) variables.

Review 5. Perspectives of the European Society of Endocrinology (ESE) and the European Society of Paediatric Endocrinology (ESPE) on rare endocrine disease.

6. Utilization of biotechnological drugs in rare diseases requiring the use of off-label drugs in children in Turkey

7. Enhancing Neurofibromatosis Clinical Trial Outcome Measures Through Patient Engagement: Lessons From REiNS.

8. Maximal Segmental Score Method for Localizing Recessive Disease Variants Based on Sequence Data.

Review 9. Research and Management of Rare Diseases in the COVID-19 Pandemic Era: Challenges and Countermeasures.

10. Childhood rare diseases and the UN convention on the rights of the child.