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Orphan Indications - Biotech, Pharma and Life Science Channel

18:28 EST 19th January 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 [223 Associated News Articles listed on BioPortfolio]

Auspex Pharmaceuticals Inc ASPX Pharmaceuticals Healthcare Deals and Alliances Profile Prices from USD $250

SummaryAuspex Pharmaceuticals Inc Auspex, a subsidiary of Teva Pharmaceutical Industries Limited is a biopharmaceutical company that develops and commercializes novel medicines for the treatment of mo...

U.S. Patent & Trademark Office (USPTO) Issues Patent To Elorac, Inc. for Treating Chronic Inflammatory Disorders

Elorac, Inc., a privately held, specialty pharmaceutical company engaged in the development of novel products for dermatology, allergy and oncology, announced that the USPTO has i...

Abeona Therapeutics Receives Orphan Drug Designation in The European Union for ABO-101 Gene Therapy in Sanfilippo Syndrome Type B

NEW YORK and CLEVELAND, Jan. 19, 2017 (GLOBE NEWSWIRE) -- ABO-101, Abeona's third AAV gene therapy program to receive EMA Orphan Designation Clinical trials anticipated to begin enrollin...

Swedish Orphan Biovitrum AB: First Patients Enrolled in 24 Month Real-world Study Evaluating Effectiveness of Elocta®

STOCKHOLM, Sweden, Jan 19, 2017 /PRNewswire/ -- Swedish Orphan Biovitrum AB (publ) (Sobi™) today announces that the first patients have been enrolled in the A-SURE study (NCT02976753).  A-S...

Regenicin Reports 2016 Operating Results, Outlines Milestones for 2017

LITTLE FALLS, N.J., Jan. 18, 2017 /PRNewswire/ -- Regenicin, Inc. (OTC Bulletin Board: RGIN), a biotechnology company specializing in the development and commercialization of regenerative cell th...

AdAlta receives Orphan Designation for its lead drug candidate targeting patients with idiopathic pulmonary disease

MELBOURNE Australia, 18 January, 2017 - AdAlta Limited (ASX: 1AD), today announced it has received Orphan Drug Designation from the United States Food and Drug Administration (FDA) for AD-114, a nov...

Tarix Orphan Receives Rare Pediatric Disease Designation for TXA127 for Recessive Dystrophic Epidermolysis Bullosa (RDEB)

CAMBRIDGE, Mass., Jan. 18, 2017 /PRNewswire/ -- Tarix Orphan LLC today announced that the U.S. Food and Drug Administration has granted a Rare Pediatric Disease (RPD) designation for the company'...

European Commission grants orphan drug designation to ProMetic's PBI-4050 drug for the treatment of Alström Syndrome

LAVAL, QC, Jan. 18, 2017 /PRNewswire/ - ProMetic Life Sciences Inc. (TSX: PLI) (OTCQX: PFSCF), ("ProMetic" or the "Corporation") announced today that its orally active lead drug candidate, PBI-40...

PhaseBio Announces $14.7 Million in Convertible Notes to Advance Clinical Development of PB1046 in Orphan Cardiopulmonary Disorders

MALVERN, Pa., Jan. 18, 2017 (GLOBE NEWSWIRE) -- PhaseBio Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company developing biopolymer-based drugs for the treatment of orphan diseases...

Remedy Pharmaceuticals’ CIRARA Granted Orphan Drug Designation For Treatment of Severe Cerebral Edema Caused By Acute Ischemic Stroke

Remedy Pharmaceuticals, a privately-held pharmaceutical company focused on bringing life-saving hospital-based treatments to people affected by central nervous system related edem...

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Companies [47 Associated Companies listed on BioPortfolio]

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€...

Lung Therapeutics, Inc.

Lung Therapeutics, Inc. is a pharmaceutical company formed to leverage 25 years of leading research in lung injury and disease by pursuing niche, orphan drug indications for which...

Ovid Therapeutics Inc.

Ovid Therapeutics Inc. is a privately-held, New York based, biopharmaceutical company committed to transforming the lives of patients with rare and orphan diseases of the brain an...

Emmaus Life Sciences, Inc.

Emmaus Life Sciences is dedicated to the discovery, development and commercialization of innovative treatments and therapies for rare diseases. The Company’s research on sick...

Clinical Trials [32 Associated Clinical Trials listed on BioPortfolio]

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.

SCLERoderma et Adipose-DErived Stroma Cells

Systemic sclerosis (SSc) is an auto-immune orphan disease mainly characterized by an alteration of the microvascular network, and by cutaneous and visceral fibrosis. Hands are frequently a...

Role of PTK-7 in Acute Myeloid Leukemias

The pseudo tyrosine kinase receptor 7 (PTK7) is an orphan tyrosine kinase receptor assigned to the planar cell polarity pathway. PTK7 is expressed in normal myeloid progenitors and CD34(+)...

PubMed Articles [108 Associated PubMed Articles listed on BioPortfolio]

Lysosomal Proteins as a Therapeutic Target in Neurodegeneration.

Several proteins that are mutated in lysosomal storage diseases are linked to neurodegenerative disease. This review focuses on some of these lysosomal enzymes and transporters, as well as current the...

Assessing the identity and expression level of the cytochrome P450 20A1 (CYP20A1) gene in the BPA-, BDE-47, and WAF-exposed copepods Tigriopus japonicus and Paracyclopina nana.

CYP20A1 is a member of the cytochrome P450 (CYP) superfamily, identified as an orphan P450 without any assigned biological function; hence, its continued status as an "orphan" gene. In order to addres...

Toward the next step in G protein-coupled receptor research: a knowledge-driven analysis for the next potential targets in drug discovery.

More than 800 G protein-coupled receptor (GPCR) genes have been discovered in the human genome. Towards the next step in GPCR research, we performed a knowledge-driven analysis of orphan class-A GPCRs...

The Hcp proteins fused with diverse extended-toxin domains represent a novel pattern of antibacterial effectors in type VI secretion systems.

The type VI secretion system (T6SS) is a widespread molecular weapon deployed by many bacterial species to target eukaryotic host cells or rival bacteria. Using a dynamic injection mechanism, diverse ...

The NR4A subfamily of nuclear receptors: potential new therapeutic targets for the treatment of inflammatory diseases.

Introduction Prolonged inflammatory response contributes to the pathogenesis of chronic disease-related disturbances. Among nuclear receptors (NRs), the orphan NR4A subfamily, which includes Nur77 (NR...

4-HYDROXYNONENAL PROTEIN ADDUCTS: KEY MEDIATOR IN RETT SYNDROME OXINFLAMMATION.

In the last 15 years a strong correlation between oxidative stress (OxS) and Rett syndrome (RTT), a rare neurodevelopmental disorder known to be caused in 95% of the cases, by a mutation in the methyl...

▼Pitolisant for narcolepsy.

▼Pitolisant (Wakix-Bioprojet Pharma) is a new treatment for adults with narcolepsy with or without cataplexy. It was licensed for use in the EU in March last year and has orphan drug status.(1) Here...

Nuclear receptor retinoid-related orphan receptor alpha promotes apoptosis but is reduced in human gastric cancer.

Retinoid-related orphan receptor α (RORα) is a nuclear receptor, which regulates inflammation and immune responses, lipid metabolism and circadian rhythm. Although RORα suppresses breast tumor inva...

Pharmaceutical lobbying in Brazil: a missing topic in the public health research agenda.

In the US, where registration of lobbyists is mandatory, the pharmaceutical industry and private health-care providers spend huge amounts of money seeking to influence health policies and government d...

Dysregulation of metallothionein and circadian genes in human hepatocellular carcinoma.

Hepatocellular carcinoma (HCC) is the major threat to human health, and disruption of circadian clock genes is implicated in hepatocarcinogenesis. This study examined the dysregulation of metallothion...

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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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