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Literature DB >> 25752159

Rare diseases and effective treatments: are we delivering?

Lucio Luzzatto¹, Carla E M Hollak², Timothy M Cox³, Arrigo Schieppati⁴, Christoph Licht⁵, Helena Kääriäinen⁶, Giampaolo Merlini⁷, Franz Schaefer⁸, Steven Simoens⁹, Luca Pani¹⁰, Silvio Garattini¹¹, Giuseppe Remuzzi¹².

Abstract

Mesh：

Year: 2015 PMID： 25752159 DOI： 10.1016/S0140-6736(15)60297-5

Source DB: PubMed Journal: Lancet ISSN： 0140-6736 Impact factor: 79.321

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13 in total

Review 1. Clinical promise of next-generation complement therapeutics.

Authors: Dimitrios C Mastellos; Daniel Ricklin; John D Lambris
Journal: Nat Rev Drug Discov Date: 2019-07-19 Impact factor: 84.694

2. Orphan drug policies and use in pediatric nephrology.

Authors: Diana Karpman; Peter Höglund
Journal: Pediatr Nephrol Date: 2016-10-13 Impact factor: 3.714

Review 3. Pharmacovigilance of medicines for rare and ultrarare diseases.

Authors: Marco Sardella; Glyn Belcher
Journal: Ther Adv Drug Saf Date: 2018-08-14

4. Novel Treatments for Rare Cancers: The U.S. Orphan Drug Act Is Delivering-A Cross-Sectional Analysis.

Authors: Clemens Stockklausner; Anette Lampert; Georg F Hoffmann; Markus Ries
Journal: Oncologist Date: 2016-03-28

5. Liver transplantation for aHUS: still needed in the eculizumab era?

Authors: Rosanna Coppo; Roberto Bonaudo; R Licia Peruzzi; Alessandro Amore; Andrea Brunati; Renato Romagnoli; Mauro Salizzoni; Miriam Galbusera; Eliana Gotti; Erica Daina; Marina Noris; Giuseppe Remuzzi
Journal: Pediatr Nephrol Date: 2015-11-24 Impact factor: 3.714

Rare diseases and effective treatments: are we delivering?

Review 1. Clinical promise of next-generation complement therapeutics.

2. Orphan drug policies and use in pediatric nephrology.

Review 3. Pharmacovigilance of medicines for rare and ultrarare diseases.

4. Novel Treatments for Rare Cancers: The U.S. Orphan Drug Act Is Delivering-A Cross-Sectional Analysis.

5. Liver transplantation for aHUS: still needed in the eculizumab era?

6. Demographics, in-hospital analysis, and prevalence of 33 rare diseases with effective treatment in Shanghai.

7. Abstracts from the 3rd International PPRI Conference, October 12-13, 2015, Vienna, Austria.

8. Negotiating prices of drugs for rare diseases.

9. Publication outcome of abstracts submitted to the American Academy of Ophthalmology meeting.

10. A model of mucopolysaccharidosis type IIIB in pigs.