当前位置： SCI文献检索 > Orphanet Journal of Rare Diseases期刊下所有文献 > Economic impact of screening for X-linked Adrenoleukodystrophy within a newborn blood spot screening programme.

Economic impact of screening for X-linked Adrenoleukodystrophy within a newborn blood spot screening programme.

Abstract：

BACKGROUND:A decision tree model was built to estimate the economic impact of introducing screening for X-linked adrenoleukodystrophy (X-ALD) into an existing tandem mass spectrometry based newborn screening programme. The model was based upon the UK National Health Service (NHS) Newborn Blood Spot Screening Programme and a public service perspective was used with a lifetime horizon. The model structure and parameterisation were based upon literature reviews and expert clinical judgment. Outcomes included health, social care and education costs and quality adjusted life years (QALYs). The model assessed screening of boys only and evaluated the impact of improved outcomes from hematopoietic stem cell transplantation in patients with cerebral childhood X-ALD (CCALD). Threshold analyses were used to examine the potential impact of utility decrements for non-CCALD patients identified by screening. RESULTS:It is estimated that screening 780,000 newborns annually will identify 18 (95%CI 12, 27) boys with X-ALD, of whom 10 (95% CI 6, 15) will develop CCALD. It is estimated that screening may detect 7 (95% CI 3, 12) children with other peroxisomal disorders who may also have arisen symptomatically. If results for girls are returned an additional 17 (95% CI 12, 25) cases of X-ALD will be identified. The programme is estimated to cost an additional £402,000 (95% CI £399-407,000) with savings in lifetime health, social care and education costs leading to an overall discounted cost saving of £3.04 (95% CI £5.69, £1.19) million per year. Patients with CCALD are estimated to gain 8.5 discounted QALYs each giving an overall programme benefit of 82 (95% CI 43, 139) QALYs. CONCLUSION:Including screening of boys for X-ALD into an existing tandem mass spectrometry based newborn screening programme is projected to reduce lifetime costs and improve outcomes for those with CCALD. The potential disbenefit to those identified with non-CCALD conditions would need to be substantial in order to outweigh the benefit to those with CCALD. Further evidence is required on the potential QALY impact of early diagnosis both for non-CCALD X-ALD and other peroxisomal disorders. The favourable economic results are driven by estimated reductions in the social care and education costs.

journal_name

Orphanet J Rare Dis

journal_title

Orphanet journal of rare diseases

authors

Bessey A,Chilcott JB,Leaviss J,Sutton A

doi

10.1186/s13023-018-0921-4

subject

Has Abstract

pub_date

2018-10-11 00:00:00

pages

179

issue

1

issn

1750-1172

pii

10.1186/s13023-018-0921-4

journal_volume

13

pub_type

杂志文章

相关文献

Orphanet Journal of Rare Diseases文献大全
  • European lipodystrophy registry: background and structure.

    abstract：BACKGROUND:Lipodystrophy syndromes comprise a group of extremely rare and heterogeneous diseases characterized by a selective loss of adipose tissue in the absence of nutritional deprivation or catabolic state. Because of the rarity of each lipodystrophy subform, research in this area is difficult and international co-...

    journal_title：Orphanet journal of rare diseases

    pub_type： 杂志文章

    doi：10.1186/s13023-020-1295-y

    authors： von Schnurbein J,Adams C,Akinci B,Ceccarini G,D'Apice MR,Gambineri A,Hennekam RCM,Jeru I,Lattanzi G,Miehle K,Nagel G,Novelli G,Santini F,Santos Silva E,Savage DB,Sbraccia P,Schaaf J,Sorkina E,Tanteles G,Vantyghem MC

    更新日期：2020-01-15 00:00:00

  • A retrospective analysis of health care utilization for patients with mitochondrial disease in the United States: 2008-2015.

    abstract：BACKGROUND:Mitochondrial disease (MD) is a heterogeneous group of disorders characterized by impaired energy production caused by abnormal oxidative phosphorylation. Diagnosis of MD is challenging given the variability in how the disease can affect an individual's neurologic, cardiovascular, ophthalmologic, or gastroen...

    journal_title：Orphanet journal of rare diseases

    pub_type： 杂志文章

    doi：10.1186/s13023-018-0949-5

    authors： Cohen B,Balcells C,Hotchkiss B,Aggarwal K,Karaa A

    更新日期：2018-11-22 00:00:00

  • Behçet's disease.

    abstract：UNLABELLED:DEFINITION OF THE DISEASE: Behçet disease (BD) is a chronic, relapsing, multisystemic disorder characterized by mucocutaneous, ocular, vascular and central nervous system manifestations. EPIDEMIOLOGY:BD seems to cluster along the ancient Silk Road, which extends from eastern Asia to the Mediterranean basin....

    journal_title：Orphanet journal of rare diseases

    pub_type： 杂志文章,评审

    doi：10.1186/1750-1172-7-20

    authors： Saadoun D,Wechsler B

    更新日期：2012-04-12 00:00:00

  • KH176 under development for rare mitochondrial disease: a first in man randomized controlled clinical trial in healthy male volunteers.

    abstract：BACKGROUND:Mitochondrial disorders are a clinically, biochemically and genetically heterogeneous group of multi-system diseases, with an unmet medical need for treatment. KH176 is an orally bio-available small molecule under development for the treatment of mitochondrial(-related) diseases. The compound is a member of ...

    journal_title：Orphanet journal of rare diseases

    pub_type： 杂志文章,随机对照试验

    doi：10.1186/s13023-017-0715-0

    authors： Koene S,Spaans E,Van Bortel L,Van Lancker G,Delafontaine B,Badilini F,Beyrath J,Smeitink J

    更新日期：2017-10-16 00:00:00

  • A new UHPLC-MS/MS method for the screening of urinary oligosaccharides expands the detection of storage disorders.

    abstract：BACKGROUND:Oligosaccharidoses are storage disorders due to enzymatic defects involved in the breakdown of the oligosaccharidic component of glycosylated proteins. The defect cause the accumulation of oligosaccharides (OS) and, depending on the lacking enzyme, results in characteristic profiles which are helpful for the...

    journal_title：Orphanet journal of rare diseases

    pub_type： 杂志文章

    doi：10.1186/s13023-020-01662-8

    authors： Semeraro M,Sacchetti E,Deodato F,Coşkun T,Lay I,Catesini G,Olivieri G,Rizzo C,Boenzi S,Dionisi-Vici C

    更新日期：2021-01-09 00:00:00

  • European principles of inhibitor management in patients with haemophilia.

    abstract：BACKGROUND:In spite of recent major advances in the understanding and treatment of inhibitor development in patients with haemophilia, multidisciplinary management of many of these patients remains suboptimal and highly heterogenous across Europe. METHODS:Following a series of multidisciplinary meetings and a review o...

    journal_title：Orphanet journal of rare diseases

    pub_type： 杂志文章,评审

    doi：10.1186/s13023-018-0800-z

    authors： Giangrande PLF,Hermans C,O'Mahony B,de Kleijn P,Bedford M,Batorova A,Blatný J,Jansone K,European Haemophilia Consortium (EHC) and the European Association for Haemophilia and Allied Disorders (EAHAD).

    更新日期：2018-04-27 00:00:00

  • Diagnosis and management of individuals with Fetal Valproate Spectrum Disorder; a consensus statement from the European Reference Network for Congenital Malformations and Intellectual Disability.

    abstract：BACKGROUND:A pattern of major and minor congenital anomalies, facial dysmorphic features, and neurodevelopmental difficulties, including cognitive and social impairments has been reported in some children exposed to sodium valproate (VPA) during pregnancy. Recognition of the increased risks of in utero exposure to VPA ...

    journal_title：Orphanet journal of rare diseases

    pub_type： 杂志文章

    doi：10.1186/s13023-019-1064-y

    authors： Clayton-Smith J,Bromley R,Dean J,Journel H,Odent S,Wood A,Williams J,Cuthbert V,Hackett L,Aslam N,Malm H,James G,Westbom L,Day R,Ladusans E,Jackson A,Bruce I,Walker R,Sidhu S,Dyer C,Ashworth J,Hindley D,Diaz G

    更新日期：2019-07-19 00:00:00

  • Differences in cardiac phenotype and natural history of laminopathies with and without neuromuscular onset.

    abstract：OBJECTIVE:To investigate differences in cardiac manifestations of patients affected by laminopathy, according to the presence or absence of neuromuscular involvement at presentation. METHODS:We prospectively analyzed 40 consecutive patients with a diagnosis of laminopathy followed at a single centre between 1998 and 2...

    journal_title：Orphanet journal of rare diseases

    pub_type： 杂志文章

    doi：10.1186/s13023-019-1245-8

    authors： Ditaranto R,Boriani G,Biffi M,Lorenzini M,Graziosi M,Ziacchi M,Pasquale F,Vitale G,Berardini A,Rinaldi R,Lattanzi G,Potena L,Martin Suarez S,Bacchi Reggiani ML,Rapezzi C,Biagini E

    更新日期：2019-11-19 00:00:00

  • Paying for the Orphan Drug System: break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments?

    abstract：:Since its enactment in 2000, the European Orphan Medicinal Products Regulation has allowed the review and approval of approaching 70 treatments for some 55 different conditions in Europe. Success does not come without a price, however. Many of these so-called "orphan drugs" have higher price points than treatments for...

    journal_title：Orphanet journal of rare diseases

    pub_type： 杂志文章

    doi：10.1186/1750-1172-7-74

    authors： Hughes-Wilson W,Palma A,Schuurman A,Simoens S

    更新日期：2012-09-26 00:00:00

  • Common disease signatures from gene expression analysis in Huntington's disease human blood and brain.

    abstract：BACKGROUND:Huntington's disease (HD) is a devastating brain disorder with no effective treatment or cure available. The scarcity of brain tissue makes it hard to study changes in the brain and impossible to perform longitudinal studies. However, peripheral pathology in HD suggests that it is possible to study the disea...

    journal_title：Orphanet journal of rare diseases

    pub_type： 杂志文章

    doi：10.1186/s13023-016-0475-2

    authors： Mina E,van Roon-Mom W,Hettne K,van Zwet E,Goeman J,Neri C,A C 't Hoen P,Mons B,Roos M

    更新日期：2016-08-01 00:00:00

  • Parents' experiences of living with, and caring for children, adolescents and young adults with Mucopolysaccharidosis (MPS).

    abstract：BACKGROUND:Many rare diseases of childhood are life-threatening and chronically debilitating, so living with a rare disease is an on-going challenge for patients and their families. MPS is one of a range of rare inherited metabolic disorders (IMDs) that come under category 3 of life-limiting conditions, where there is ...

    journal_title：Orphanet journal of rare diseases

    pub_type： 杂志文章

    doi：10.1186/s13023-016-0521-0

    authors： Somanadhan S,Larkin PJ

    更新日期：2016-10-10 00:00:00

  • Carglumic acid enhances rapid ammonia detoxification in classical organic acidurias with a favourable risk-benefit profile: a retrospective observational study.

    abstract：BACKGROUND:Isovaleric aciduria (IVA), propionic aciduria (PA) and methylmalonic aciduria (MMA) are inherited organic acidurias (OAs) in which impaired organic acid metabolism induces hyperammonaemia arising partly from secondary deficiency of N-acetylglutamate (NAG) synthase. Rapid reduction in plasma ammonia is requir...

    journal_title：Orphanet journal of rare diseases

    pub_type： 杂志文章,多中心研究

    doi：10.1186/s13023-016-0406-2

    authors： Valayannopoulos V,Baruteau J,Delgado MB,Cano A,Couce ML,Del Toro M,Donati MA,Garcia-Cazorla A,Gil-Ortega D,Gomez-de Quero P,Guffon N,Hofstede FC,Kalkan-Ucar S,Coker M,Lama-More R,Martinez-Pardo Casanova M,Molina A,Picha

    更新日期：2016-03-31 00:00:00

  • A guide to writing systematic reviews of rare disease treatments to generate FAIR-compliant datasets: building a Treatabolome.

    abstract：BACKGROUND:Rare diseases are individually rare but globally affect around 6% of the population, and in over 70% of cases are genetically determined. Their rarity translates into a delayed diagnosis, with 25% of patients waiting 5 to 30 years for one. It is essential to raise awareness of patients and clinicians of exis...

    journal_title：Orphanet journal of rare diseases

    pub_type： 杂志文章

    doi：10.1186/s13023-020-01493-7

    authors： Atalaia A,Thompson R,Corvo A,Carmody L,Piscia D,Matalonga L,Macaya A,Lochmuller A,Fontaine B,Zurek B,Hernandez-Ferrer C,Rheinard C,Gómez-Andrés D,Desaphy JF,Schon K,Lohmann K,Jennings MJ,Synofzik M,Riess O,Yaou RB,

    更新日期：2020-08-12 00:00:00

  • LC3, an autophagosome marker, is expressed on oligodendrocytes in Nasu-Hakola disease brains.

    abstract：BACKGROUND:Nasu-Hakola disease (NHD) is a rare autosomal recessive disorder characterized by sclerosing leukoencephalopathy and multifocal bone cysts, caused by a loss-of-function mutation of either DAP12 or TREM2. TREM2 and DAP12 constitute a receptor/adaptor signaling complex expressed exclusively on osteoclasts, den...

    journal_title：Orphanet journal of rare diseases

    pub_type： 杂志文章

    doi：10.1186/1750-1172-9-68

    authors： Satoh J,Motohashi N,Kino Y,Ishida T,Yagishita S,Jinnai K,Arai N,Nakamagoe K,Tamaoka A,Saito Y,Arima K

    更新日期：2014-05-01 00:00:00

  • Clinical and endocrine characteristics and genetic analysis of Korean children with McCune-Albright syndrome: a retrospective cohort study.

    abstract：BACKGROUND:McCune-Albright syndrome (MAS) is a rare disease defined by the triad of fibrous dysplasia (FD), café au lait spots, and peripheral precocious puberty (PP). Because of the rarity of this disease, only a few individuals with MAS have been reported in Korea. We describe the various clinical and endocrine manif...

    journal_title：Orphanet journal of rare diseases

    pub_type： 杂志文章

    doi：10.1186/s13023-016-0496-x

    authors： Cho EK,Kim J,Yang A,Ki CS,Lee JE,Cho SY,Jin DK

    更新日期：2016-08-09 00:00:00

  • Pharmaceutical pricing, cost containment and new treatments for rare diseases in children.

    abstract：:Cost-containment in healthcare spending has become a central issue in public policy and healthcare reform, especially as the affordable care act adds millions of people to public and private insurance rolls. In this climate, longstanding criticism of pharmaceutical pricing has grown sharper, and many in both policy an...

    journal_title：Orphanet journal of rare diseases

    pub_type： 杂志文章,评审

    doi：10.1186/s13023-014-0152-2

    authors： Stella P,Gold-von Simson G

    更新日期：2014-10-28 00:00:00

  • New insight into clinical heterogeneity and inheritance diversity of FBLN5-related cutis laxa.

    abstract：BACKGROUND:FBLN5-related cutis laxa (CL) is a rare disorder that involves elastic fiber-enriched tissues and is characterized by lax skin and variable systemic involvement such as pulmonary emphysema, arterial involvement, inguinal hernias, hollow viscus diverticula and pyloric stenosis. This type of CL follows mostly ...

    journal_title：Orphanet journal of rare diseases

    pub_type： 杂志文章

    doi：10.1186/s13023-021-01696-6

    authors： Gharesouran J,Hosseinzadeh H,Ghafouri-Fard S,Jabbari Moghadam Y,Ahmadian Heris J,Jafari-Rouhi AH,Taheri M,Rezazadeh M

    更新日期：2021-01-28 00:00:00

  • Large homozygous RAB3GAP1 gene microdeletion causes Warburg micro syndrome 1.

    abstract：:Warburg micro syndrome (WARBM) is a genetic heterogeneous disease characterized by microcephaly, intellectual disability, brain, ocular, and endocrine anomalies. WARBM1-4 can be caused by biallelic mutations of the RAB3GAP1 (RAB3 GTPase-activating protein 1), RAB3GAP2, RAB18 (RAS-associated protein RAB18), or TBC1D20 ...

    journal_title：Orphanet journal of rare diseases

    pub_type： 信件

    doi：10.1186/s13023-014-0113-9

    authors： Picker-Minh S,Busche A,Hartmann B,Spors B,Klopocki E,Hübner C,Horn D,Kaindl AM

    更新日期：2014-10-21 00:00:00

  • Sickle cell disease induces resistance to cutaneous carcinogenesis.

    abstract：BACKGROUND:While skin carcinomas are reported in chronic ulcers and in patients treated with hydroxyurea (HU) for myeloproliferative neoplasms, no skin carcinoma has been reported in patients with sickle cell disease (SCD), presenting chronic skin ulcers or treated with HU. The objective was to estimate the risk of cut...

    journal_title：Orphanet journal of rare diseases

    pub_type： 杂志文章

    doi：10.1186/s13023-020-1341-9

    authors： Soutou B,Senet P,Lionnet F,Habibi A,Aractingi S

    更新日期：2020-03-06 00:00:00

  • The expanding spectrum of rare monogenic autoinflammatory diseases.

    abstract：:Monogenic autoinflammatory diseases are a group of hereditary disorders characterized by a clinical and biological inflammatory syndrome in which there is little or no evidence of autoimmunity. The discovery of the first causative gene in 1997 was rapidly followed by the identification of many others from the same gro...

    journal_title：Orphanet journal of rare diseases

    pub_type： 杂志文章,评审

    doi：10.1186/1750-1172-8-162

    authors： Touitou I,Galeotti C,Rossi-Semerano L,Hentgen V,Piram M,Koné-Paut I,CeRéMAI, French reference center for autoinflammatory diseases.

    更新日期：2013-10-16 00:00:00

  • Hamartomatous polyposis syndromes: a review.

    abstract：:Hamartomatous Polyposis Syndromes (HPS) are genetic syndromes, which include Peutz-Jeghers syndrome, Juvenile polyposis syndrome, PTEN hamartoma tumour syndrome (Cowden Syndrom, Bannayan-Riley-Ruvalcaba and Proteus Syndrome) as well as hereditary mixed polyposis syndrome. Other syndromes such as Gorlin Syndrome and mu...

    journal_title：Orphanet journal of rare diseases

    pub_type： 杂志文章,评审

    doi：10.1186/1750-1172-9-101

    authors： Jelsig AM,Qvist N,Brusgaard K,Nielsen CB,Hansen TP,Ousager LB

    更新日期：2014-07-15 00:00:00

  • Lower plasma cholesterol, LDL-cholesterol and LDL-lipoprotein subclasses in adult phenylketonuria (PKU) patients compared to healthy controls: results of NMR metabolomics investigation.

    abstract：BACKGROUND:Phenylketonuria (PKU; OMIM#261600) is a rare metabolic disorder caused by mutations in the phenylalanine hydroxylase (PAH) gene resulting in high phenylalanine (Phe) in blood and brain. If not treated early this results in intellectual disability, behavioral and psychiatric problems, microcephaly, motor defi...

    journal_title：Orphanet journal of rare diseases

    pub_type： 杂志文章

    doi：10.1186/s13023-020-1329-5

    authors： Cannet C,Pilotto A,Rocha JC,Schäfer H,Spraul M,Berg D,Nawroth P,Kasperk C,Gramer G,Haas D,Piel D,Kölker S,Hoffmann G,Freisinger P,Trefz F

    更新日期：2020-02-27 00:00:00

  • Juvenile xanthogranuloma as a new type of skin lesions in tuberous sclerosis complex.

    abstract：OBJECTIVE:Tuberous sclerosis complex (TSC) is a rare autosomal dominant genetic disease with many manifestations, and it involves any organ. In this study, we report a TSC patient with new type skin lesions. METHODS:A 7-month-old TSC boy with multiple cutaneous nodules was admitted in our hospital. We collected the cl...

    journal_title：Orphanet journal of rare diseases

    pub_type： 杂志文章

    doi：10.1186/s13023-020-01396-7

    authors： Lu Q,Shi XY,Wang YY,Zhang MN,Wang WZ,Wang J,Wang QH,Chen HM,Zou LP

    更新日期：2020-06-12 00:00:00

  • Impact of Friedreich's Ataxia on health-care resource utilization in the United Kingdom and Germany.

    abstract：BACKGROUND:Friedreich's Ataxia (FRDA) is a neurodegenerative disorder that causes progressive damage to the central and peripheral nervous systems having a significant impact upon quality of life. With little information in the literature, cross-sectional observational studies were conducted in the UK and Germany to co...

    journal_title：Orphanet journal of rare diseases

    pub_type： 杂志文章

    doi：10.1186/1750-1172-8-38

    authors： Giunti P,Greenfield J,Stevenson AJ,Parkinson MH,Hartmann JL,Sandtmann R,Piercy J,O'Hara J,Casas LR,Smith FM

    更新日期：2013-02-28 00:00:00

  • Nerve ultrasound characterizes AMN polyneuropathy as inhomogeneous and focal hypertrophic.

    abstract：OBJECTIVE:High-resolution nerve ultrasound (HRUS) is a painless tool to quickly evaluate peripheral nerve morphology in vivo. This study set out to characterize peripheral nerve involvement in X-linked adrenomyeloneuropathy (AMN) by HRUS. METHODS:Thirteen adults with genetically proven AMN were examined using the Ultr...

    journal_title：Orphanet journal of rare diseases

    pub_type： 杂志文章

    doi：10.1186/s13023-018-0939-7

    authors： Rattay TW,Just J,Röben B,Hengel H,Schüle R,Synofzik M,Söhn AS,Winter N,Dammeier N,Schöls L,Grimm A

    更新日期：2018-11-03 00:00:00

  • Clinical outcome, biochemical and therapeutic follow-up in 14 Austrian patients with Long-Chain 3-Hydroxy Acyl CoA Dehydrogenase Deficiency (LCHADD).

    abstract：BACKGROUND:LCHADD is a long-fatty acid oxidation disorder with immediate symptoms and long-term complications. We evaluated data on clinical status, biochemical parameters, therapeutic regimens and outcome of Austrian LCHADD patients. STUDY DESIGN:Clinical and outcome data including history, diagnosis, short- and long...

    journal_title：Orphanet journal of rare diseases

    pub_type： 杂志文章

    doi：10.1186/s13023-015-0236-7

    authors： Karall D,Brunner-Krainz M,Kogelnig K,Konstantopoulou V,Maier EM,Möslinger D,Plecko B,Sperl W,Volkmar B,Scholl-Bürgi S

    更新日期：2015-02-22 00:00:00

  • Single point mutation in Rabenosyn-5 in a female with intractable seizures and evidence of defective endocytotic trafficking.

    abstract：BACKGROUND:We report a 6.5 year-old female with a homozygous missense mutation in ZFYVE20, encoding Rabenosyn-5 (Rbsn-5), a highly conserved multi-domain protein implicated in receptor-mediated endocytosis. The clinical presentation includes intractable seizures, developmental delay, microcephaly, dysostosis, osteopeni...

    journal_title：Orphanet journal of rare diseases

    pub_type： 杂志文章

    doi：10.1186/s13023-014-0141-5

    authors： Stockler S,Corvera S,Lambright D,Fogarty K,Nosova E,Leonard D,Steinfeld R,Ackerley C,Shyr C,Au N,Selby K,van Allen M,Vallance H,Wevers R,Watkins D,Rosenblatt D,Ross CJ,Conibear E,Wasserman W,van Karnebeek C

    更新日期：2014-09-20 00:00:00

  • The alliance between genetic biobanks and patient organisations: the experience of the telethon network of genetic biobanks.

    abstract：BACKGROUND:Rare diseases (RDs) are often neglected because they affect a small percentage of the population (6-8 %), which makes research and development of new therapies challenging processes. Easy access to high-quality samples and associated clinical data is therefore a key prerequisite for biomedical research. In t...

    journal_title：Orphanet journal of rare diseases

    pub_type： 杂志文章

    doi：10.1186/s13023-016-0527-7

    authors： Baldo C,Casareto L,Renieri A,Merla G,Garavaglia B,Goldwurm S,Pegoraro E,Moggio M,Mora M,Politano L,Sangiorgi L,Mazzotti R,Viotti V,Meloni I,Pellico MT,Barzaghi C,Wang CM,Monaco L,Filocamo M

    更新日期：2016-10-24 00:00:00

  • Patient reported outcome measures in a large cohort of patients with type 1 Gaucher disease.

    abstract：BACKGROUND:It is now acknowledged that the input of patients in health outcome assessment is vital to understanding the impact of diseases and interventions for those diseases. This study is the first report of patient-reported outcome measures (PROM) in a large cohort of patients with type 1 Gaucher disease (GD1) enab...

    journal_title：Orphanet journal of rare diseases

    pub_type： 杂志文章

    doi：10.1186/s13023-020-01544-z

    authors： Dinur T,Istaiti M,Frydman D,Becker-Cohen M,Szer J,Zimran A,Revel-Vilk S

    更新日期：2020-10-13 00:00:00

  • In vitro and in vivo therapeutic approach for a small cell carcinoma of the ovary hypercalcaemic type using a SCCOHT-1 cellular model.

    abstract：BACKGROUND:The small cell ovarian carcinoma of the hypercalcemic type (SCCOHT) which preferably affects young women during regenerative age represents a rare and aggressive form of ovarian tumors with poor prognosis and lacks an efficient therapy. METHODS AND RESULTS:In vitro chemotherapy testing in a fluorescence ass...

    journal_title：Orphanet journal of rare diseases

    pub_type： 杂志文章

    doi：10.1186/s13023-014-0126-4

    authors： Otte A,Rauprich F,Hillemanns P,Park-Simon TW,von der Ohe J,Hass R

    更新日期：2014-08-08 00:00:00