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Orphan Indications - Biotech, Pharma and Life Science Channel

05:58 EDT 24th May 2017 | BioPortfolio

Examples of Ophan Drug Indications include:

Anaemia, sickle cell
Cystic fibrosis (CF)
Duchenne muscular dystrophy
Glioma
Graft vs host disease (GvHD)
Hepatoma, liver cancer
Hodgkin Lymphoma
Leukaemia, acute lymphocytic (ALL)
Leukaemia, acute myeloid (AML)
Leukaemia, chronic lymphocytic (CLL)
Leukaemia, chronic myeloid (CML)
Multiple myeloma
Myelodysplastic syndrome
Non-Hodgkin lymphoma (NHL)
Ovarian cancer
Pancreatic cancer
Pulmonary fibrosis, idiopathic
Pulmonary hypertension
Renal cell carcinoma (RCC)
Soft tissue sarcoma

News Articles [257 Associated News Articles listed on BioPortfolio]

Neuway Pharma GmbH Pharmaceuticals Healthcare Deals and Alliances Profile [Updated: 10052017] Prices from USD $250

SummaryNeuway Pharma GmbH Neuway focuses on the preclinical and clinical development of therapeutics for the treatment of central nervous system diseases. Its proprietary CNS drug delivery platform fa...

Palladio Biosciences Formed to Develop Transformative Medicines for Orphan Diseases of the Kidney

--Repositioning Lixivaptan for Polycystic Kidney Disease-- Palladio Biosciences, Inc. (Palladio) http://palladiobio.com/, a private biopharmaceutical company founded to develo...

Global Duchenne Muscular Dystrophy Therapeutics Market By Drug, By Therapeutic Approach Clinical Trial Assessment & Pipeline Analysis Outlook 2022

LONDON, May 23, 2017 /PRNewswire/ -- Duchenne Muscular Dystrophy (DMD) is a fatal genetic disorders diagnosed in children around the world. DMD is often referred to as orphan or rare diseases, as it a...

Orphan Drug Commercialisation and Market Access in Emerging Markets [Updated: 01062016] Prices from USD $2195

Market access for orphan drugs: Can your company break through in emerging markets?Find out which emerging markets offer the best investment prospects for orphan drug manufacturers.Based on interviews...

Dompé Receives Positive CHMP Opinion in Europe For Oxervate® (Cenegermin Eye Drops) For the Treatment of Adult Patients with Moderate or Severe Neurotrophic Keratitis

Designed to treat moderate or severe neurotrophic keratitis in adults, if approved by the European Commission, Oxervate® would be the first biotechnological treatmen...

BioAxone BioSciences CEO Lisa McKerracher Invited Speaker at the 12th Annual Neurotech Investing and Partnering Conference, Orphan Disease Panel

BioAxone BioSciences, Inc., a clinical-stage biotechnology company focused on developing innovative drugs to restore neurological function, today announced that Lisa McKerr...

viDA Therapeutics Inc Pharmaceuticals Healthcare Deals and Alliances Profile [Updated: 18052017] Prices from USD $250

SummaryviDA Therapeutics Inc viDA focuses on the discovery and development of therapeutics for the treatment of autoimmune and chronic inflammatory diseases. The company's lead product candidate VTI10...

Ammonett Pharma Receives Positive Opinion from European Orphan Medicinal Products Committee for MK-0677 for Treatment of Pediatric Growth Hormone Deficiency

MIDLOTHIAN, Va., May 23, 2017 (GLOBE NEWSWIRE) -- Ammonett Pharma LLC (the Company or Ammonett), a privately-held biotechnology company developing a novel, daily, orally-administered mini-pill Gr...

Orphan Drug Commercial Models: Sustaining the rare disease business [Updated: 01052016] Prices from USD $695

Choosing the right commercial model for orphan drug successThe societal and commercial ambitions of orphan drug developers determine which commercial model will best meet their aims. Profit, potentia...

Amryt Pharma plc AMYT Financial and Strategic SWOT Analysis Review [Updated: 15052017] Prices from USD $300

SummaryAmryt Pharma plc Amryt is a specialty pharmaceutical company, which focuses on the development, manufacture and commercialization of novel treatments for patients with rare and orphan diseases....

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Companies [51 Associated Companies listed on BioPortfolio]

Palladio Biosciences, Inc.

Palladio Biosciences is a privately-owned, clinical stage biopharmaceutical company developing medicines for orphan diseases of the kidney and is located in Newtown, PA. For more ...

Regenacy Pharmaceuticals, LLC

Regenacy Pharmaceuticals, LLC is a clinical-stage biopharmaceutical company regenerating biological function by protein acetylation for the treatment of peripheral neuropathies, c...

Santhera Pharmaceuticals Holding Ltd

Santhera Pharmaceuticals is a Swiss biopharmaceutical company focusing on the discovery, development and marketing of small molecule pharmaceutical products for the treatment of neuromuscular diseases...

Imara Inc.

Imara Inc., a Cydan Development company, is dedicated to developing novel therapeutics for patients with sickle cell disease. Imara is developing IMR-687, a highly s...

Pharnext SAS

Pharnext is an advanced clinical stage biopharmaceutical company founded by renowned scientists and entrepreneurs including Professor Daniel Cohen, a pioneer in modern genomics. P...

Regenacy Pharmaceuticals

Regenacy Pharmaceuticals, LLC is a clinical-stage biopharmaceutical company regenerating biological function by protein acetylation for the treatment peripheral neuropathies, cogn...

Auven Therapeutics

Auven Therapeutics is a private equity company focused on the healthcare industry. The Company’s strategy is to acquire controlling ownership in promising therapeutic products or platforms. ...

Atlantic Healthcare plc

Atlantic Healthcare is an international specialty pharmaceutical company focussed on diseases of the gastrointestinal tract and Inflammatory Bowel Disease (IBD). The Company's lea...

Millendo Therapeutics, Inc.

Millendo Therapeutics is focused on developing a portfolio of disease-modifying treatments for endocrine diseases caused by hormone dysregulation. Our product candidates seek to i...

Atlantic Healthcare

Atlantic Healthcare is an international specialty pharmaceutical company focussed on diseases of the gastrointestinal tract and Inflammatory Bowel Disease (IBD). The Company's lea...

Clinical Trials [35 Associated Clinical Trials listed on BioPortfolio]

Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype A

This is an open, Phase I / II clinical trial to evaluate the safety and efficacy of a hematopoietic gene therapy procedure with an orphan drug consisting of a lentiviral vector carrying th...

The LD Lync Study - Natural History Study of Genetic Lipodystrophy Syndromes

Genetic lipodystrophy syndromes are extremely rare, orphan diseases with overall estimated prevalence of less than 2,000 in the United States. These rare disorders characterized by selecti...

An Open Label, Single-dose, Single Period ADME Study of A4250 in Healthy Subjects

The primary objectives of the study are to assess the mass balance recovery after a single dose of carbon-14 [14C]-A4250 as a capsule and to provide plasma, urine and faecal samples for me...

Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study

: Epidermolysis bullosa (EB) simplex is a rare orphan disease caused by a mutation in DNA leading to abnormal dominant keratins in the skin. Patients with EB simplex develop lifelong painf...

A Study of GSK2981278 Ointment in Subjects With Plaque Psoriasis

GSK2981278 is an inverse agonist of retinoic acid receptor-related orphan receptor (ROR) gamma. The aim of this study is to evaluate the safety, tolerability, clinical effect, and systemic...

CD180 Overexpression in Follicular Lymphoma

Background:Altered Toll-like receptor (TLR) expression levels and/or mutations in its signaling pathway (such as MyD88 mutation) contribute to the pathogenesis of lymphoproliferative disor...

A Safety and Pharmakokinetic Study of A4250 Alone or in Combination With A3384

The primary objectives of the study are to evaluate the safety, tolerability and pharmacokinetics of A4250 after single or multiple oral doses in healthy subjects. In addition, will evalua...

Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study

Epidermolysis bullosa (EB) simplex is a rare orphan disease caused by a mutation in DNA leading to abnormal dominant keratins in the skin. Patients with EB simplex develop lifelong painful...

Compare Train and 3D-4D Left Ventricular Systolic Function in Subjects Suffering From Dystrophy and Healthy Subjects

Steinert's disease is an orphan disease. The prognosis of patients with this disease is conditioned by cardiac involvement. Search an early stage alterations in contractile function in sub...

Clinical Phase II Trial to Evaluate CD34+ Cells Mobilization and Collection in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene. FANCOSTEM-1

Fanconi anemia (FA) is a congenital disease characterized by bone marrow failure and increased incidence of malignant tumors. The Project pursue the optimization of the collection of hemat...

PubMed Articles [116 Associated PubMed Articles listed on BioPortfolio]

Drugs for Children.

The "Therapeutic Orphan" status of children is gradually improving, but our efforts to eliminate off-label/off-evidence use must be sustained for safer and more effective pediatric drug therapy. This ...

Studies in Rare Diseases: A Descriptive Analysis of Completed Orphan Drug Benefit Assessments at the Federal Joint Committee.

Ovary-specific depletion of the nuclear receptor Nr5a2 compromises expansion of the cumulus oophorus but not fertilization by ICSI.

The orphan nuclear receptor, liver receptor homolog-1 (Nr5a2) is widely expressed in mammalian tissues, its ovarian expression is restricted to granulosa cells of activated follicles. We employed the ...

Large-scale data-driven integrative framework for extracting essential targets and processes from disease-associated gene data sets.

Populations worldwide currently face several public health challenges, including growing prevalence of infections and the emergence of new pathogenic organisms. The cost and risk associated with drug ...

The Roles of Orexins in Sleep/Wake Regulation.

Orexin A and orexin B are hypothalamic neuropeptides initially identified as endogenous ligands for two orphan G-protein coupled receptors (GPCRs). A deficiency of orexin signaling results in the slee...

Single-Center Experience with Intimal Sarcoma, an Ultra-Orphan, Commonly Fatal Mesenchymal Malignancy.

Characterization of NMB, GRP and their receptors (BRS3, NMBR and GRPR) in chickens.

The two structurally and functionally related peptides, gastrin-releasing peptide (GRP) and neuromedin B (NMB) play critical roles in many physiological/pathological processes in mammals. However, the...

Ternary complex of human RORγ ligand-binding domain, inverse agonist and SMRT peptide shows a unique mechanism of corepressor recruitment.

Retinoid-related orphan receptor gamma (RORγ) directly controls the differentiation of Th17 cell and the production of interleukin-17, which plays an integral role in autoimmune diseases. To obtain i...

Detecting Gravitational Wave Memory without Parent Signals.

Gravitational-wave memory manifests as a permanent distortion of an idealized gravitational-wave detector and arises generically from energetic astrophysical events. For example, binary black hole mer...

The Molecular Revolution in Cutaneous Biology: Emerging Landscape in Genomic Dermatology: New Mechanistic Ideas, Gene Editing, and Therapeutic Breakthroughs.

Stunning technological advances in genomics have led to spectacular breakthroughs in the understanding of the underlying defects, biological pathways and therapeutic targets of skin diseases leading ...

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Medical and Biotech [MESH] Definitions

An orphan nuclear receptor that is implicated in regulation of steroidogenic pathways. It is unlike most orphan nuclear receptors in that it appears to lack an essential DNA-binding domain and instead acts as a transcriptional co-repressor. Mutations in the gene Dax-1 cause congenital adrenal hypoplasia.

A family of cell surface receptors that were originally identified by their structural homology to neurotropic TYROSINE KINASES and referred to as orphan receptors because the associated ligand and signaling pathways were unknown. Evidence for the functionality of these proteins has been established by experiments showing that disruption of the orphan receptor genes results in developmental defects.

A DNA-binding orphan nuclear receptor that has specificity for directly repeated (DR) AGGTCA sequences. It binds DNA as either as a homodimer or as a heterodimer with the closely-related orphan nuclear receptor NUCLEAR RECEPTOR SUBFAMILY 2, GROUP C, MEMBER 2. The protein was originally identified as a PROSTATE-specific protein and is involved in the regulation of variety of cellular processes, including CELL DIFFERENTIATION; CELL PROLIFERATION; and APOPTOSIS.

An orphan nuclear receptor that has specificity for hormone response elements found in the promoters of target genes. It binds DNA either as a homodimer or as heterodimer with the closely-related orphan nuclear receptor NUCLEAR RECEPTOR SUBFAMILY 2, GROUP C, MEMBER 1. The protein was originally identified as a TESTES-specific protein and is involved in the regulation of variety of cellular processes, including CELL DIFFERENTIATION; CELL PROLIFERATION; and APOPTOSIS.

Production of drugs or biologicals which are unlikely to be manufactured by private industry unless special incentives are provided by others.

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