Expert Opinion on Orphan Drugs
Overview
Expert Opinion on Orphan Drugs is a scientific journal, published by Informa Healthcare since 2013 in English. The journal's country of origin is United Kingdom.
Details
Details
Abbr.
Expert Opin Orphan Drugs
Publisher
Informa Healthcare
Start
2013
End
2024
Frequency
Monthly
e-ISSN
2167-8707
Country
United Kingdom
Language
English
Metrics
Metrics
h-index / Ranks: 10997
27
SJR / Ranks: 13573
199
CiteScore / Ranks: 11103
1.80
JIF / Ranks: 7093
0.8
Recent Articles
1.
Miller K, Kraft S, Ipe A, Fermaglich L
Expert Opin Orphan Drugs
. 2022 May;
9(11-12):265-272.
PMID: 35548705
Background: The Orphan Drug Act was created to stimulate the development of drugs and biologics for rare diseases. Investigating products that have received orphan drug designation provide a greater understanding...
2.
Muchtar E, Gertz M
Expert Opin Orphan Drugs
. 2021 Sep;
5(8):655-663.
PMID: 34567849
Introduction: The field of systemic amyloidosis is experiencing major advances in diagnostic and prognostic methods coupled with a growing availability in treatment options. Areas Covered: Treatment of AL amyloidosis traditionally...
3.
Han R, Fry L, Kantor A, McClements M, Xue K, MacLaren R
Expert Opin Orphan Drugs
. 2021 May;
9(1):13-24.
PMID: 34040899
Introduction: Choroideremia is an X-linked inherited retinal degeneration resulting from mutations in the gene, encoding Rab escort protein-1 (REP1), a protein regulating intracellular vesicular transport. Loss-of-function mutations in lead to...
4.
Rosenberg J, Chen A, Kaminsky S, Crystal R, Sondhi D
Expert Opin Orphan Drugs
. 2020 Dec;
7(11):473-500.
PMID: 33365208
Areas Covered: The reader will be introduced to the NCL subtypes, natural histories, experimental animal models, and biomarkers for NCL progression; challenges and different therapeutic approaches, and the latest pre-clinical...
5.
Thompson W, Mondal G, VanLith C, Kaiser R, Lillegard J
Expert Opin Orphan Drugs
. 2020 Nov;
8(7):245-256.
PMID: 33224636
Introduction: Inborn errors of metabolism (IEMs) often result from single-gene mutations and collectively cause liver dysfunction in neonates leading to chronic liver and systemic disease. Current treatments for many IEMs...
6.
Bose P
Expert Opin Orphan Drugs
. 2020 Oct;
7(10):415-425.
PMID: 33094033
Introduction: The Janus kinase (JAK)1/2 inhibitor ruxolitinib provides rapid, sustained and often dramatic benefits to patients with myelofibrosis, inducing spleen shrinkage and ameliorating symptoms, and improves survival. However, the drug...
7.
Bradford D, Reilly K, Widemann B, Sandler A, Kummar S
Expert Opin Orphan Drugs
. 2020 Aug;
4(1):93-103.
PMID: 32765971
Introduction: Rare tumors account for one fourth of adult tumors; in children, rare tumors represent approximately 15-20% of childhood malignancies, thus accounting for a significant burden of disease. The rarity...
8.
Menghini M, Cehajic-Kapetanovic J, MacLaren R
Expert Opin Orphan Drugs
. 2020 Apr;
8(2-3):67-78.
PMID: 32231889
Introduction: Retinitis pigmentosa (RP) is the most common form of inherited retinal degenerations with an estimated prevalence of 1 in 4,000 and more than 1 million individuals affected worldwide. With...
9.
10.
Pacifici M
Expert Opin Orphan Drugs
. 2019 Aug;
6(6):385-391.
PMID: 31448184
Introduction: Hereditary multiple exostoses (HME) is a rare congenital pediatric disorder characterized by osteochondromas forming next to the growth plates in young patients. The osteochondromas cause multiple health problems that...