Advertisement

Topics

Orphan Indications - Biotech, Pharma and Life Science Channel

18:38 EDT 28th June 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 [278 Associated News Articles listed on BioPortfolio]

FDA Grants Profounda Inc. Orphan Drug Designation for treatment of Granulomatous Amebic Encephalitis (GAE) with Miltefosine

ORLANDO, Fla., June 28, 2017 /PRNewswire/ -- Profounda, Inc. ("Profounda") announced today that it has received the US Food and Drug Administration's Orphan Drug Designation for the...

Resverlogix Announces Fourth Positive Recommendation From Data Safety Monitoring Board For Phase 3 Study of Apabetalone

A data review by the independent Data Safety Monitoring Board resulted in a recommendation for the BETonMACE trial to continue as planned without any modification. CALGARY, June 28, 2017 /PRNewswi...

EMA Grants Orphan Designation to Edison Pharmaceuticals for EPI-743 for Treatment of Leigh Syndrome

MOUNTAIN VIEW, Calif., Sept. 12, 2012 /PRNewswire/ -- Edison Pharmaceuticals announced today that the Committee for Orphan Medicinal Products (COMP), European Medicines Agency, has grante...

Regenacy Pharmaceuticals Appoints Simon S. Jones as President and Chief Executive Officer

-- Former CEO Walter Ogier to Remain on Company’s Board -- Regenacy Pharmaceuticals, LLC, a clinical-stage biopharmaceutical company that is regenerating biological f...

TP Therapeutics Announces FDA Orphan Drug Designation Granted to TPX-0005 for Treatment of Non-Small Cell Lung Adenocarcinomas Harboring ALK, ROS1, or NTRK Oncogenic Rearrangements

TP Therapeutics, Inc., a privately held, clinical-stage biopharmaceutical company focusing on addressing oncology drug resistance, announced today that U.S. Food and Drug Administ...

Survey Shows Intensifying Focus on Cost and Demonstration of Value in Increasingly Competitive Orphan Drug Space

BURLINGTON, Mass., June 27, 2017 /PRNewswire/ -- Decision Resources Group finds that more than 40% of surveyed U.S. physicians perceive an increase over the last three years in the degree of paye...

EMA Approves the Potential to Dose Every 14 Days or Longer in Updated Dosing Regimen for Alprolix®

STOCKHOLM, June 27, 2017 /PRNewswire/ -- Swedish Orphan Biovitrum AB (publ) (Sobi™) has received approval from the European Medicines Agency (EMA) to update the dosing information for Alprolix® (ef...

NeuroVive and Yungjin Pharm Start Clinical Development in Genetic Mitochondrial Disease

LUND, Sweden and SEOUL, South Korea, June 27, 2017 /PRNewswire/ -- NeuroVive Pharmaceutical AB (Nasdaq Stockholm: NVP, OTCQX: NEVPF) and Yungjin Pharm Corporation Ltd...

Questcor Teams with Child Neurology Foundation (CNF) to Continue Funding Infantile Spasms Scientific Research

HAYWARD, Calif., Oct. 27, 2011 /PRNewswire/ -- Questcor Pharmaceuticals, Inc. (NASDAQ: QCOR) announced that it is again partnering with the Child Neurology Foundation (CNF) to fund a $30,000 grant to ...

Telik Announces Orphan Designation Of Telintra® For Treatment Of Myelodysplastic Syndrome

PALO ALTO, Calif., Jan. 11, 2013 /PRNewswire/ -- Telik, Inc. (Nasdaq: TELK) was notified today that its product candidate, ezatiostat HCL (Telintra), has been granted orphan drug designati...

Events [0 Results]

None

Companies [53 Associated Companies listed on BioPortfolio]

Genexine, Inc.

Genexine, Inc., listed on KOSDAQ (095700) since 2009, is a leading biotherapeutics company focused on immuno-oncology and orphan disease. Genexine has robust pipelines in clinical...

Rare Disease Communications

Rare Disease Communications® is a healthcare communications and media company focused on the rare disease community and dedicated to raising awareness of rare diseases and orph...

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...

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 [114 Associated PubMed Articles listed on BioPortfolio]

Correction to "The Orphan Receptor GPR17 Is Unresponsive to Uracil Nucleotides and Cysteinyl Leukotrienes".

microRNA cluster 106a~363 is involved in T-Helper 17 (Th17) cell differentiation.

T-helper cell 17 (Th17) mediated inflammation is associated with various diseases including autoimmune encephalitis, inflammatory bowel disease and lung diseases such as chronic obstructive pulmonary ...

Protective effects of astragaloside IV against ovalbumin-induced allergic rhinitis are mediated by T-box protein expressed in T cells/GATA-3 and forkhead box protein 3/retinoic acid-related orphan nuclear receptor γt.

3-O-β-D-xylopyranosyl-6-O-β-D-glucopyranosyl-cycloastragenol, or Astragaloside IV (AST), is one of the major active ingredients isolated from Astragalus membranaceous with distinct pharmacological e...

Caspase-2: an orphan enzyme out of the shadows.

Caspase-2 has been embodied as an initiator or executioner protease in diverse apoptotic scenarios. However, accumulating evidence is challenging this view, pertaining to its true role. The enzyme's c...

Epithelial Gpr116 regulates pulmonary alveolar homeostasis via Gq/11 signaling.

Pulmonary function is dependent upon the precise regulation of alveolar surfactant. Alterations in pulmonary surfactant concentrations or function impair ventilation and cause tissue injury. Identific...

Retinoic acid-related orphan receptor alpha (RORA) variants and risk of breast cancer.

Breast cancer is the most common type of cancer and the second leading cause of cancer death in females. Despite numerous studies in this field, the etiology and clinical behavior of breast tumors hav...

Orphan diseases - on the way to better patient care.

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 ...

Structural Basis of the Human Endoglin-BMP9 Interaction: Insights into BMP Signaling and HHT1.

Endoglin (ENG)/CD105 is an essential endothelial cell co-receptor of the transforming growth factor β (TGF-β) superfamily, mutated in hereditary hemorrhagic telangiectasia type 1 (HHT1) and involve...

Development and validation of an ultra-performance liquid chromatography-tandem mass spectrometry method for quantification of SR1001, an inverse agonist of retinoid-related orphan receptors, and its application to pharmacokinetic studies in streptozotocin-induced diabetic mice.

Retinoic acid receptor-related orphan receptors (RORs) play critical roles in the onset and progression of type I diabetes, an autoimmune disease characterized by the destruction of pancreatic β-cell...

Videos

None available.

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.

Quick Search
Advertisement
 

Relevant Topics

Pharmacy
Pharmacy is the science and technique of preparing as well as dispensing drugs and medicines. It is a health profession that links health sciences with chemical sciences and aims to ensure the safe and effective use of pharmaceutical drugs. The scope of...

Pulmonary
Pulmonary relating to or associated with the lungs eg Asthma, chronic bronchitis, emphysema, COPD, Cystic Fibrosis, Influenza,  Lung Cancer, Pneumonia, Pulmonary Arterial Hypertension, Sleep Disorders etc Follow and track Lung Cancer News ...

review and buy Orphan Indications market research data and corporate reports here

Channels Quicklinks