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Orphan Indications - Biotech, Pharma and Life Science Channel

19:07 EDT 20th August 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 [277 Associated News Articles listed on BioPortfolio]

InterMune to Release Second Quarter Financial Results on August 6

BRISBANE, Calif., July 28, 2014 /PRNewswire/ -- InterMune, Inc. (NASDAQ: ITMN) today announced that it will release its second quarter 2014 financial results at the close of the U.S. markets on W...

Leading Biotech Companies Receiving Orphan Drug Designation in 2017

LAS VEGAS, August 17, 2017 /PRNewswire/ -- Under the Orphan Drug Act (ODA), drugs, vaccines, and diagnostic agents qualify for orphan status if they are intended to treat a disease affecting less ...

Protalex Awarded Grant from FDA Office of Orphan Products Development to Foster Clinical Development of PRTX-100 for the Treatment of Immune Thrombocytopenia

Protalex, Inc. (OTCQB:PRTX), a clinical-stage biopharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development (OO...

Halloran Consulting Group Announces Role as US Regulatory Lead for Curtana Pharmaceuticals’ CT-179 Orphan Drug Designation for the Treatment of Gliomas

Halloran Consulting Group, a national consultancy serving biotech, pharma, & medical device companies with clinical, quality and regulatory compliance projects, today a...

US FDA grants Lion TCR two orphan drug designations of T cell therapy against Hepatocellular Carcinoma

SINGAPORE, Aug. 16, 2017 /PRNewswire/ -- US FDA has granted two orphan drug designations (ODD) for T cell therapy products to Singapore-based biotech company, Lion TCR Pte Ltd, for the treatment of he...

Concordia International Corp CXR Financial and Strategic SWOT Analysis Review [Report Updated: 07082017] Prices from USD $300

SummaryConcordia International Corp Concordia, formerly Concordia Healthcare Corp is a healthcare products provider that offers orphan drugs. The company undertakes the management and acquisition of l...

Market Access Impact (EU5) [Haemophilia A]

LONDON, Aug. 14, 2017 /PRNewswire/ -- Losing share to competitors? Understanding market barriers can help close the gapHaemophilia A treatment is a highly competitive market in the EU5 countries,...

H3 Biomedicine Granted Orphan Drug Designation of H3B-8800 for Treatment of Acute Myelogenous Leukemia and Chronic Myelomonocytic Leukemia

H3 Biomedicine Inc., a clinical stage biopharmaceutical company specializing in the discovery and development of precision medicines for oncology and a member of Eisai’s global ...

Cerecor Inc. Announces Retirement of Dr. Uli Hacksell as President and Chief Executive Officer

BALITIMORE, MD--(Marketwired - August 14, 2017) - Cerecor Inc. (NASDAQ: CERC), a clinical-stage biopharmaceutical company developing innovative drug candidates for patients with neurologic and neurop...

Cerecor Inc. Reports Second Quarter 2017 Financial Results

BALTIMORE, MD--(Marketwired - August 14, 2017) - Cerecor Inc. (NASDAQ: CERC), a biopharmaceutical company developing innovative drug candidates for patients with neurologic and neuropsychiatric disor...

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Companies [55 Associated Companies listed on BioPortfolio]

Cavion, Inc.

Cavion, Inc. is a privately held clinical stage biotechnology company creating therapies modulating the T-type calcium channel (Cav3) for the treatment of neurological diseases. C...

Can-Fite BioPharma Ltd.

Can-Fite BioPharma Ltd. (NYSE MKT: CANF) (TASE: CFBI) is an advanced clinical stage drug development Company with a platform technology that is designed to address multi-billion d...

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...

Clinical Trials [36 Associated Clinical Trials listed on BioPortfolio]

PARP-1 Inhibition in Pulmonary Arterial Hypertension : a Pilot Study

The main OBJECTIVE of this proposal is to extend our preclinical findings on the role of DNA damage and poly(ADP-ribose) polymerases (PARP) inhibition as a therapy for a devastating diseas...

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...

PubMed Articles [116 Associated PubMed Articles listed on BioPortfolio]

Anti-Dll4 Antibody Inhibits the Differentiation of Th17 Cells in Asthmatic Mice.

T helper 17 (Th17) cells play an important role in allergic asthma, and the Notch ligand Delta-like ligand (Dll)4 has been reported to direct the differentiation of Th17 cells. In this study, experime...

Protein misfolding, amyotrophic lateral sclerosis and guanabenz: protocol for a phase II RCT with futility design (ProMISe trial).

Recent studies suggest that endoplasmic reticulum stress may play a critical role in the pathogenesis of amyotrophic lateral sclerosis (ALS) through an altered regulation of the proteostasis, the cell...

A fourth subtype of retinoic acid receptor-related orphan receptors is activated by oxidized all-trans retinoic acid in medaka (Oryzias latipes).

The three known subtypes of the retinoic acid receptor-related orphan receptor (ROR) have been implicated in the control of immunity, brain function, and circadian rhythm in mammals. Here, we demonstr...

Pharmacology and drug development in rare diseases: the attractiveness and expertise of the French medical pharmacology.

Developing drugs for rare disease can be challenging due to specific rare disease characteristics. The French Medical Pharmacology is structured and positioned to play a major role in Orphan Drug Rese...

Nuclear orphan receptor NR2F6 as a safeguard against experimental murine colitis.

Nuclear receptors are known to regulate both immune and barrier functions in the GI tract. The nuclear orphan receptor NR2F6 has been shown to suppress the expression of proinflammatory cytokines in T...

Age dependent plasticity in endocannabinoid modulation of pain processing through postnatal development.

Significant age and experience-dependent remodelling of spinal and supraspinal neural networks occur resulting in altered pain responses in early life. In adults endogenous opioid peptide and endocann...

Anaesthesia and orphan disease: Management of a case of Strumpell-Lorrain disease and review of the literature.

Anaesthesia and orphan disease series: What is the yield?

RORγt and RORα signature genes in human Th17 cells.

RORγt and RORα are transcription factors of the RAR-related orphan nuclear receptor (ROR) family. They are expressed in Th17 cells and have been suggested to play a role in Th17 differentiation. Alt...

High expression levels of Wnt5a and Ror2 in laryngeal squamous cell carcinoma are associated with poor prognosis.

The present study investigated the prognostic significance of Wnt family member 5a (Wnt5a) and receptor tyrosine kinase-like orphan receptor 2 (Ror2) expression in laryngeal squamous cell carcinoma (L...

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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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