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Orphan Indications - Biotech, Pharma and Life Science Channel

15:03 EDT 24th April 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 [260 Associated News Articles listed on BioPortfolio]

Swedish Orphan Biovitrum AB SOBI Pharmaceuticals Healthcare Deals and Alliances Profile [Updated: 22032017] Prices from USD $250

SummarySwedish Orphan Biovitrum AB Sobi, formerly Biovitrum AB, is an integrated biopharmaceutical company that focuses on the development of products for the treatment of rare diseases. The company s...

News and press releases: First medicine for spinal muscular atrophy

Orphan medicine Spinraza recommended by CHMP under accelerated assessment

Onxeo Receives the EnterNext Tech 40 Label

Onxeo is admitted to the “Tech 40” index, which incorporates 40 outstanding small and midcap Tech companies listed on the various Euronext markets in Europe Regulat...

Protagonist Therapeutics Announces PTG-200 Data Accepted for Oral Presentation at Digestive Diseases Week

MILPITAS, Calif., April 20, 2017 /PRNewswire/ -- Protagonist Therapeutics, Inc. (Nasdaq: PTGX) today announced that preclinical research findings on PTG-200, the company's oral peptide IL-23 rece...

Xenetic Biosciences’ PolyXen™ Platform Technology Accepted for Poster Presentation at the 13th Annual Protein Engineering Summit (PEGS) Boston

Xenetic Biosciences, Inc. (NASDAQ: XBIO) (“Xenetic” or the “Company”), a clinical-stage biopharmaceutical company focused on the discovery, research and development...

Gamida Cell Announces Publication of Positive Clinical Outcomes from NiCord-Transplanted Patients in the Journal of Biology of Blood and Marrow Transplantation

JERUSALEM, April 20, 2017 /PRNewswire/ -- Gamida Cell, a leader in cellular and immune therapies for the treatment of cancer and orphan genetic diseases, announced today the publication of positi...

Eyevensys Receives Approval From the UK Medicines and Healthcare Products Regulatory Agency to Advance its EyeCET Platform Into Clinical Development

PARIS, April 20, 2017 /PRNewswire/ -- Eyevensys, a private biotechnology company developing its proprietary EyeCET platform, the first non-viral gene expression technology that enables the safe, l...

Aldeyra Therapeutics, Inc. Receives Orphan Drug Designation from the U.S. Food and Drug Administration for ADX-102 in Sjögren-Larsson Syndrome

LEXINGTON, MA -- (Marketwired) -- 04/20/17 -- Aldeyra Therapeutics, Inc. (NASDAQ: ALDX) (Aldeyra), a clinical-stage biotech company devoted to treating diseases thought to be related to endogenous al...

Cantex Pharmaceuticals to Present Overview of CX-02 at the World Orphan Drug Congress

WESTON, Fla., April 19, 2017 /PRNewswire/ -- Cantex Pharmaceuticals Inc., a clinical stage biopharmaceutical company developing proprietary pharmaceuticals that improve the treatment of cancers a...

Catabasis Pharmaceuticals to Present at the American Academy of Neurology 69th Annual Meeting

Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that it will present data from Part A and Part B of the MoveDMD t...

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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 [108 Associated PubMed Articles listed on BioPortfolio]

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 ...

Digoxin Attenuates Murine Experimental Colitis by Downregulating Th17-related Cytokines.

Digoxin, a cardiac glycoside used for the treatment of heart failure, was reported to inhibit the retinoid-related orphan receptor gamma t (RORγt) and attenuate the severity of experimental autoimmun...

In-Silico Drug discovery approach targeting receptor tyrosine kinase-like orphan receptor 1 for cancer treatment.

Receptor tyrosine kinases (RTK) are important cell signaling molecules that influence many cellular processes. Receptor tyrosine kinase such as orphan receptor 1 (Ror1), a surface antigen, is a member...

Diversity of integrative and conjugative elements of Streptococcus salivarius and their intra- and interspecies transfer.

Integrative and Conjugative Elements (ICEs) are widespread chromosomal mobile genetic elements, which can transfer autonomously by conjugation in bacteria. Thirteen ICEs with a conjugation module clos...

Information ranks highest: Expectations of female adolescents with a rare genital malformation towards health care services.

Access to highly specialized health care services and support to meet the patient's specific needs is critical for health outcome, especially during age-related transitions within the health care syst...

Compositional differences among female-associated and embryo-associated microbiota of the viviparous Pacific Beetle cockroach, Diploptera punctata.

All cockroach species, except one, harbor the endosymbiont Blattabacterium, transmitted from females to embryos. Adult cockroaches acquire non-Blattabacterium bacteria as part of their gut microbiota ...

Molecular phenotyping of transient postnatal tyrosine hydroxylase neurons in the rat bed nucleus of the stria terminalis.

The bed nucleus of the stria terminalis (BNST) is a complex integrative centre in the forebrain, composed of multiple sub-nuclei, each with discrete populations of neurons. Progress in understanding B...

Thermal Hall Effect in a Phonon-Glass Ba_{3}CuSb_{2}O_{9}.

A distinct thermal Hall signal is observed in a quantum spin liquid candidate Ba_{3}CuSb_{2}O_{9}. The transverse thermal conductivity shows a power-law temperature dependence below 50 K, where a spi...

Celastrol-Induced Nur77 Interaction with TRAF2 Alleviates Inflammation by Promoting Mitochondrial Ubiquitination and Autophagy.

Mitochondria play an integral role in cell death, autophagy, immunity, and inflammation. We previously showed that Nur77, an orphan nuclear receptor, induces apoptosis by targeting mitochondria. Here,...

Comparative transcriptomics of Entelegyne spiders (Araneae, Entelegynae), with emphasis on molecular evolution of orphan genes.

Next-generation sequencing technology is rapidly transforming the landscape of evolutionary biology, and has become a cost-effective and efficient means of collecting exome information for non-model o...

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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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