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Orphan Indications - Biotech, Pharma and Life Science Channel

13:26 EDT 25th March 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 [254 Associated News Articles listed on BioPortfolio]

Concordia International Corp CXR Pharmaceuticals Healthcare Deals and Alliances Profile [Updated: 23022017] Prices from USD $250

SummaryConcordia International Corp Concordia, formerly Concordia Healthcare Corp is a pharmaceutical company that offers orphan drugs. The company undertakes the management and acquisition of legacy ...

FDA Provides Cornerstone Pharmaceuticals Approval to Initiate Pivotal Study for CPI-613, its Metabolism-Directed Anticancer Compound

CRANBURY, N.J., March 23, 2017 /PRNewswire/ -- Cornerstone Pharmaceuticals, Inc., a clinical-stage, oncology-focused pharmaceutical company, announced a major milestone today.  Following suc...

Gamida Cell Receives Additional EMA Orphan Drug Designation for NiCord® in Haematopoietic Stem Cell Transplantation (HSCT)

JERUSALEM, March 23, 2017 /PRNewswire/ -- Gamida Cell, a leader in cellular and immune therapies for the treatment of cancer and orphan genetic diseases, announced today that orphan drug designat...

Eiger BioPharmaceuticals Reports Fourth Quarter and Full Year 2016 Financial Results

PALO ALTO, Calif., March 23, 2017 /PRNewswire/ -- Eiger BioPharmaceuticals, Inc. (Nasdaq: EIGR), focused on the development and commercialization of targeted therapies for rare diseases, announce...

viDA Therapeutics Inc Pharmaceuticals Healthcare Deals and Alliances Profile [Updated: 27022017] 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...

Moleculin Receives Orphan Drug Designation for Annamycin for the Treatment of Acute Myeloid Leukemia

HOUSTON, TX--(Marketwired - March 22, 2017) - Moleculin Biotech, Inc., (NASDAQ: MBRX) ("Moleculin" or the "Company"), a preclinical pharmaceutical company focused on the development of anti-cancer dr...

Onxeo to Present Data Supporting Three Key Orphan Oncology Assets at AACR Annual Meeting

Preclinical studies of AsiDNATM, Livatag® and Beleodaq® Regulatory News: Onxeo S.A. (Paris:ONXEO) (NASDAQ OMX:ONXEO) (Euronext Paris, NASDAQ Copenhage...

Eagle Pharmaceuticals Announces Three New Patents Issued for Bendeka

Bendeka protected by 14 patents running from 2026 through 2033 Thirteen Orange Book listed patents Eagle Pharmaceuticals, Inc. (Nasdaq:EGRX) (“Eagle” or “the Co...

Gene Therapy for Rare Diseases: Challenges and opportunities encountered

LONDON, March 21, 2017 /PRNewswire/ -- Throughout the past decade, the orphan drug landscape has undergone a considerable amount of maturing. As a result, an increasing amount of orphan drugs are bein...

NantKwest Announces FDA Grant of Orphan Drug Designation for the Company’s aNK Natural Killer Cell Therapy in Merkel Cell Carcinoma

NantKwest, Inc. (Nasdaq:NK), a pioneering, next generation, clinical-stage immunotherapy company focused on harnessing the unique power of our immune system using natural killer (...

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Companies [50 Associated Companies listed on BioPortfolio]

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...

Cambridge BioMarketing

Cambridge BioMarketing is an orphan disease and specialty market agency, blending medical insights and award-winning creativity with deep experience to build some of the worldâ€...

Clinical Trials [34 Associated Clinical Trials listed on BioPortfolio]

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...

Etiology of Orphan Community-based Meningitis and Meningo-encephalitis.

The primary purpose of this study is to assess the contribution of a non-invasive sampling (pharyngeal swab) in the diagnosis of community based meningitis or meningo-encephalitis.

PubMed Articles [103 Associated PubMed Articles listed on BioPortfolio]

Deletion of a Histone Acetyltransferase Leads to the Pleiotropic Activation of Natural Products in Metarhizium robertsii.

Histone deacetylation normally decreases the gene expression in organisms. By genome-wide deletions of epigenetic regulators in entomopathogenic fungus Metarhizium robertsii, unexpected activations of...

The International Hypothermia Registry (IHR): Dieter's ESAO Winter Schools and Beat's International Hypothermia Registry.

Accidental hypothermia could be listed as an 'orphan disease,' since mild hypothermia is common but has no severe medical consequences, whereas severe hypothermia is rare and life-threatening. In orde...

Drugs in clinical development for the treatment of amyotrophic lateral sclerosis.

Amyotrophic Lateral Sclerosis (ALS) is a fatal motor neuron progressive disorder for which no treatment exists to date. However, there are other investigational drugs and therapies currently under cli...

Optogenetic methods in drug screening: technologies and applications.

The optogenetic revolution enabled spatially-precise and temporally-precise control over protein function, signaling pathway activation, and animal behavior with tremendous success in the dissection o...

Anaesthesia and orphan disease: Difficult ventilation following intubation in Goldenhar syndrome.

Anaesthesia and orphan diseases: Airway management in Proteus disease.

Anaesthesia and orphan disease: A child with Congenital Cataract Facial Dysmorphism neuropathy (CCFDN) syndrome: a case report.

Anaesthesia and orphan disease: Rapid sequence induction in systemic mastocytosis.

Small-Molecule RORγt Antagonists: One Stone Kills Two Birds.

Although small-molecule thymus-specific isoform of retinoic acid receptor-related orphan nuclear receptor γ (RORγt) antagonists suppressing interleukin (IL)-17-producing T helper (Th17) cells are wi...

Identification of Arabidopsis Genic and Non-genic Promoters by Paired-end Sequencing of TSS Tags.

Information about transcription start sites (TSSs) provides foundational data for analyses of promoter architecture. In this report, we used paired- and single-end deep sequencing to analyze Arabidops...

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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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