Catalyst Pharma sees net price of drug, once free, topping $300,000

Catalyst Pharmaceuticals Inc said on Monday that it expects its drug for a rare disease, which had long been available to patients for free, will cost more than $300,000 per year including rebates to insurers and other discounts.
Source: Reuters: Health - Category: Consumer Health News Tags: healthNews Source Type: news

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Krabbe disease is a rare neurological disorder caused by a deficiency in the lysosomal enzyme, β-galactocerebrosidase, resulting in demyelination of the central and peripheral nervous systems. If left without ...
Source: Orphanet Journal of Rare Diseases - Category: Internal Medicine Authors: Tags: Research Source Type: research
Since incentives were introduced to promote orphan drugs in Europe, several dozens of drugs have been registered at the European level. However, patient access on a national level remains very heterogeneous ac...
Source: Orphanet Journal of Rare Diseases - Category: Internal Medicine Authors: Tags: Research Source Type: research
Severe mucopolysaccharidosis type I (also known as Hurler syndrome) is a rare devasting recessive genetic disease caused by the deficiency of an enzyme. Hematopoietic stem cell transplant is the standard of ca...
Source: Orphanet Journal of Rare Diseases - Category: Internal Medicine Authors: Tags: Research Source Type: research
Publication date: Available online 15 February 2019Source: The Journal of Allergy and Clinical Immunology: In PracticeAuthor(s): Markus G. Seidel, Gerhard Kindle, Benjamin Gathmann, Isabella Quinti, Matthew Buckland, Joris van Montfrans, Raphael Scheible, Stephan Rusch, Lukas M. Gasteiger, Bodo Grimbacher, Nizar Mahlaoui, Stephan Ehl, Michael Albert, Sarah Beaussant Cohen, Jacinta Bustamante, Andrew Cant, Jean-Laurent Casanova, Helen Chapel, Genevieve de Saint Basile, Esther de VriesAbstractPatient registries are instrumental for clinical research in rare diseases. They help to achieve a sufficient sample size for epidemio...
Source: The Journal of Allergy and Clinical Immunology: In Practice - Category: Allergy & Immunology Source Type: research
This study was a retrospective and descriptive of LT cases diagnosed in pathology laboratories in Senegal during five years (2011-2015). The LT was found in 9 cases. The mean age at diagnosis was 41.9 years (18/58) without sex predominance (sex-ratio = 0.8). Tobacco intoxication (3/9), tuberculosis contagion (1/9), HIV immunodepression (2/9), and diabetes (1/9) were the various risk factors found. Dysphonia was a constant symptom (9/9) associated with dysphagia (2/9) and cervical adenopathy (1/9). The macroscopic presentation was tumoral-like (7/9) and erythematosus and fibrinoid (2/9). The LT was of glottic seat...
Source: Bulletin de la Societe de Pathologie Exotique - Category: Tropical Medicine Tags: Bull Soc Pathol Exot Source Type: research
The male breast encompasses a number of benign and malignant breast conditions. The commonest cause of male breast enlargement is gynaecomastia. This can be physiological, idiopathic or pathological. Male breast cancer (MBC) is a rare disease comprising less than 1% of all breast cancers. The commonest histology is grade 2 ductal no special type carcinoma. Unlike the female breast, papillary in situ and invasive carcinomas are not uncommon in the male breast. MBC is often of the luminal A phenotype, similar to the post menopausal female breast cancer.
Source: Diagnostic Histopathology - Category: Pathology Authors: Tags: Mini-symposium: breast pathology Source Type: research
Publication date: 2019Source: European Journal of Radiology Open, Volume 6Author(s): P. Lomoro, I. Simonetti, G. Vinci, V. Fichera, L. Tarotto, P. Trovato, M.S. Prevedoni GoroneAbstractLangerhans cell histiocytosis (LCH) is a rare disease of the myeloid precursor cells, it predominantly occurs in the skull and long bones as unifocal bone lesions. Aneurysmal bone cysts (ABC) are benign, expansive and lytic bone. Reports of secondary ABC occurring in LCH are rare, having only been reported twice in the skull. Here, we report the first case of LCH masquerading as ABC in a 14-month-old female child who presented with a rapidly...
Source: European Journal of Radiology Open - Category: Radiology Source Type: research
Contributors : Nadav Ahituv ; Jay Shendure ; Martin KircherSeries Type : OtherOrganism : Homo sapiens ; Mus musculusThe majority of common variants associated with common diseases, as well as an unknown proportion of causal mutations for rare diseases, fall in noncoding regions of the genome. Although catalogs of noncoding regulatory elements are steadily improving, we have a limited understanding of the functional effects of mutations within them. Here, we performed saturation mutagenesis in conjunction with massively parallel reporter assays on 20 disease-associated gene promoters and enhancers, generating functional mea...
Source: GEO: Gene Expression Omnibus - Category: Genetics & Stem Cells Tags: Other Homo sapiens Mus musculus Source Type: research
In phenylketonuria, casein glycomacropeptide (CGMP) requires modification with the addition of some essential and semi essential amino acids to ensure suitability as a protein substitute. The optimal amount an...
Source: Orphanet Journal of Rare Diseases - Category: Internal Medicine Authors: Tags: Research Source Type: research
Cornelia de Lange syndrome (CdLS) and Rubinstein-Taybi syndrome (RSTS) are both rare congenital multiple malformation disorders caused by genes associated with transcription. They share a number of similar fea...
Source: Orphanet Journal of Rare Diseases - Category: Internal Medicine Authors: Tags: Research Source Type: research
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