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Orphan Indications - Biotech, Pharma and Life Science Channel

06:59 EST 23rd February 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 [233 Associated News Articles listed on BioPortfolio]

Bellicum Presents Updated Clinical Results at 2017 BMT Tandem Meetings on BPX-501 in Orphan Inherited Blood Disorders and Hematologic Cancers

HOUSTON, Feb. 22, 2017 (GLOBE NEWSWIRE) -- Bellicum Pharmaceuticals, Inc. (Nasdaq:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood...

Immune Design Receives Orphan Drug Designation for G100 Intratumoral Product Candidate

SEATTLE and SOUTH SAN FRANCISCO, Calif., Feb. 22, 2017 (GLOBE NEWSWIRE) -- Immune Design (Nasdaq:IMDZ), a clinical-stage immunotherapy company focused on oncology, today announced that the U.S. Foo...

BioVie Submits Additional Information to FDA for BIV201 Clinical Trial in Ascites Patients

BEVERLY, MA--(Marketwired - February 22, 2017) - BioVie Inc. (OTC PINK: BIVI), a development-stage company focused on the discovery, development, and commercialization of innovative drug therapies fo...

Lysogene Receives Orphan Drug Designation from EMA for LYS-GM101 for Treatment of GM1 Gangliosidosis

Regulatory News: Lysogene, (Paris:LYS) (the “Company” FR0013233475 – LYS) a leading, clinical-stage biotechnology company specializing in gene therapy for ra...

Eagle Pharmaceuticals Announces New Patents Issued for Bendeka

-- Bendeka protected by 10 patents running from 2026 through 2033 – -- Total of nine Orange Book listed patents – -- Additional four patents allowed -- ...

Innovate Biopharmaceuticals To Highlight Its Late-Stage Product Pipeline Targeting Celiac Disease And Ulcerative Colitis At The Cowen Healthcare Conference On March 6, 2017

RALEIGH, N.C., Feb. 21, 2017 /PRNewswire/ -- Innovate Biopharmaceuticals Inc., a leader in developing innovative therapies for autoimmune and inflammatory diseases with significant unmet needs in...

Concordia International Corp CXR Financial and Strategic SWOT Analysis Review [Updated: 13012017] Prices from USD $300

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 ...

Sobi™ Enters Into new Distribution Agreement With Valeant for Ammonul®

STOCKHOLM, Feb 21, 2017 /PRNewswire/ -- Swedish Orphan Biovitrum AB (publ) (Sobi™) today announces that the company has entered into a 3-year agreement with Valeant Pharmaceuticals Ireland for t...

NeuroVive Pharmaceutical AB Year End Report January - December 2016

STOCKHOLM, Feb 21, 2017 /PRNewswire/ -- Important steps forward and new research collaborations Business operations Important events October – December 2016 •   The devel...

Soricimed Publishes the Results of Phase I Study of SOR-C13 in Patients with Advanced Solid Tumors

TORONTO, ONTARIO -- (Marketwired) -- 02/21/17 -- Soricimed Biopharma Inc. ("Soricimed"), a clinical-stage pharmaceutical company discovering and developing peptide-based cancer therapeutics, is plea...

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Companies [48 Associated Companies listed on BioPortfolio]

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€...

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...

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 [119 Associated PubMed Articles listed on BioPortfolio]

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.

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...

How do Kenyan orphan girls experience less meaningful lives and how much does it matter for 'health'?

Currently, 2.5 million orphaned children are living in Kenya and 56 million orphaned children are living across sub-Saharan Africa. No empirical research has investigated meaningfulness of life amon...

Prioritizing Rare Diseases.

Measuring societal preferences for rarity has been proposed to determine whether paying premium prices for orphan drugs is acceptable.

Influence of Tiopronin on the Metabolism of Alcohol in Healthy Subjects.

Drug safety- and drug-alcohol interaction studies have mainly been conducted for frequently prescribed drugs with high financial interests. Orphan drugs such as tiopronin (ORPHA25073) are often neglec...

Congenital Cataract in Gpr161vl/vl Mice Is Modified by Proximal Chromosome 15.

The morphology and severity of human congenital cataract varies even among individuals with the same mutation, suggesting that genetic background modifies phenotypic penetrance. The spontaneous mouse ...

Cytokine changes in response to TPO receptor agonist treatment in primary immune thrombocytopenia.

Thrombopoietin receptor agonists (TPO-RAs) have been clinically used in primary immune thrombocytopenia (ITP) with favorable outcomes, while their effect on cytokine regulation in ITP remains unknown....

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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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