PubMed

Expensive drugs for rare disorders: to treat or not to treat? The case of enzyme replacement therapy for mucopolysaccharidosis VI.

Schlander M, Beck M.

Curr Med Res Opin. 2009 May;25(5):1285-93. Review.PMID: 19366306 [PubMed - indexed for MEDLINE]Related articles

Treatment options for HUS secondary to Escherichia coli O157:H7.

Bitzan M.

Kidney Int Suppl. 2009 Feb;(112):S62-6. Review.PMID: 19180140 [PubMed - indexed for MEDLINE]Related articles

A unifying strategy for clinical trials in rare urological malignancy.

Nicholson S.

Nat Clin Pract Urol. 2008 Dec;5(12):652-3. Epub 2008 Nov 18. Review. No abstract available. PMID: 19015645 [PubMed - indexed for MEDLINE]Related articles

International perspectives on the cost-effectiveness of tandem mass spectrometry for rare metabolic conditions.

Norman R, Haas M, Wilcken B.

Health Policy. 2009 Mar;89(3):252-60. Epub 2008 Sep 26. Review.PMID: 18823674 [PubMed - indexed for MEDLINE]Related articles

Rare childhood diseases: how should we respond?

Zurynski Y, Frith K, Leonard H, Elliott E.

Arch Dis Child. 2008 Dec;93(12):1071-4. Epub 2008 Aug 6. Review.PMID: 18684747 [PubMed - indexed for MEDLINE]Related articles

Optimal therapy for rare disorders and genetic diseases: ethical and political challenges.

MacLeod S.

Proc West Pharmacol Soc. 2007;50:21-3. Review.PMID: 18605224 [PubMed - indexed for MEDLINE]Related articles

Gene therapy of inherited diseases.

Fischer A, Cavazzana-Calvo M.

Lancet. 2008 Jun 14;371(9629):2044-7. Review. No abstract available. PMID: 18555917 [PubMed - indexed for MEDLINE]Related articles

Why rare diseases are an important medical and social issue.

Schieppati A, Henter JI, Daina E, Aperia A.

Lancet. 2008 Jun 14;371(9629):2039-41. Review. No abstract available. PMID: 18555915 [PubMed - indexed for MEDLINE]Related articles

Unusual tumors of the thyroid gland.

Baloch ZW, LiVolsi VA.

Endocrinol Metab Clin North Am. 2008 Jun;37(2):297-310, vii. Review.PMID: 18502328 [PubMed - indexed for MEDLINE]Related articles

Developing a national collaborative study system for rare genetic diseases.

Watson MS, Epstein C, Howell RR, Jones MC, Korf BR, McCabe ER, Simpson JL.

Genet Med. 2008 May;10(5):325-9.PMID: 18496030 [PubMed - indexed for MEDLINE]Related articles

The Greig cephalopolysyndactyly syndrome.

Biesecker LG.

Orphanet J Rare Dis. 2008 Apr 24;3:10. Review.PMID: 18435847 [PubMed - indexed for MEDLINE]Related articlesFree article

Efficient ways exist to obtain the optimal sample size in clinical trials in rare diseases.

van der Lee JH, Wesseling J, Tanck MW, Offringa M.

J Clin Epidemiol. 2008 Apr;61(4):324-30. Epub 2008 Feb 21. Review.PMID: 18313556 [PubMed - indexed for MEDLINE]Related articles

Networking for rare diseases: a necessity for Europe.

Aymé S, Schmidtke J.

Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz. 2007 Dec;50(12):1477-83. Review.PMID: 18026888 [PubMed - indexed for MEDLINE]Related articles

Impacts of national surveillance for uncommon conditions in childhood.

Zurynski YA, Peadon E, Bower C, Elliott EJ.

J Paediatr Child Health. 2007 Nov;43(11):724-31. Review.PMID: 17924937 [PubMed - indexed for MEDLINE]Related articles

A patient and family perspective on gene therapy for rare diseases.

Kent A, Oosterwijk C.

J Gene Med. 2007 Oct;9(10):922-3. Review.PMID: 17880044 [PubMed - indexed for MEDLINE]Related articles

Autoinflammatory syndromes with a dermatological perspective.

Kanazawa N, Furukawa F.

J Dermatol. 2007 Sep;34(9):601-18. Review.PMID: 17727363 [PubMed - indexed for MEDLINE]Related articles

Access to information supporting availability of medicines for patients suffering from rare diseases looking for possible treatments: the EuOrphan Service.

Stakisaitis D, Spokiene I, Juskevicius J, Valuckas KP, Baiardi P.

Medicina (Kaunas). 2007;43(6):441-6. Review.PMID: 17637514 [PubMed - indexed for MEDLINE]Related articlesFree article

Inhibitor development in haemophilia B: an orphan disease in need of attention.

DiMichele D.

Br J Haematol. 2007 Aug;138(3):305-15. Review.PMID: 17614818 [PubMed - indexed for MEDLINE]Related articles

Rare and real illnesses that affect our students.

Ilardi D.

School Nurse News. 2007 Mar;24(2):16-21. Review. No abstract available. PMID: 17393820 [PubMed - indexed for MEDLINE]Related articles

Evidence-based medicine for rare diseases: implications for data interpretation and clinical trial design.

Behera M, Kumar A, Soares HP, Sokol L, Djulbegovic B.

Cancer Control. 2007 Apr;14(2):160-6. Review.PMID: 17387301 [PubMed - indexed for MEDLINE]Related articlesFree article