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Orphan Indications - Biotech, Pharma and Life Science Channel

19:02 EDT 21st October 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 [165 Associated News Articles listed on BioPortfolio]

North America Injectable Drug Delivery Market Industry Trends and Forecast to 2024 [Report Updated: 01072017] Prices from USD $4200

Synopsis: North America injectable drug delivery market is expected to reach USD 354.5 billion by 2024 from USD 154.0 billion in 2016, at a CAGR of 11.0% in the forecast period 2017 to 2024. The repor...

VBL Therapeutics Announces Orphan Drug Designation for VB-111 in Europe

New EMA orphan approval for Ovarian Cancer complements previously granted VB-111 orphan status for Glioblastoma in the US and Europe TEL AVIV, Israel, Oct. 20, 2017 (GLOBE NEWSWIRE) -- VBL Ther...

Immune Design Announces G100’s Receipt of Orphan Drug Designation by the EMA for the Treatment of Follicular Non-Hodgkin’s Lymphoma

SEATTLE and SOUTH SAN FRANCISCO, Calif., Oct. 19, 2017 (GLOBE NEWSWIRE) -- Immune Design (Nasdaq:IMDZ), a clinical-stage immunotherapy company focused on oncology, today announced that the Europe...

FDA Grants Pluristem Orphan Drug Designation for Its PLX-R18 Cell Therapy as Treatment for Acute Radiation Syndrome

HAIFA, Israel, Oct. 19, 2017 (GLOBE NEWSWIRE) -- Pluristem Therapeutics Inc. (NASDAQ:PSTI) (TASE:PSTI), a leading developer of placenta-based cell therapy products, announced today that the U.S. ...

Lysogene Announces Company Presentations at Upcoming Scientific Conferences

Regulatory News: Lysogene (Paris:LYS) (FR0013233475 – LYS), a leading, biopharmaceutical company pioneering gene therapy technologies to treat central nervous sy...

Cerecor, Inc. Regains Compliance with NASDAQ Minimum Bid Price Requirement

BALTIMORE, MD--(Marketwired - October 18, 2017) - Cerecor, Inc. (NASDAQ: CERC), a biopharmaceutical company developing innovative drug candidates for patients with neurologic and neuropsychiatric dis...

viDA Therapeutics Inc Pharmaceuticals Healthcare Deals and Alliances Profile [Report Updated: 12102017] Prices from USD $250

SummaryviDA Therapeutics Inc viDA is a research institute that concentrates on the discovery and development of therapeutics for the treatment of autoimmune and chronic inflammatory diseases. The inst...

Trinity Partners Launches New Whitepaper to Assess Value in Ultra-Orphan Markets

Explores the crucial clinical and commercial attributes that drive success for ultra-orphan, non-oncology products Trinity Partners, a leading global life sciences c...

Global Orphan Diseases Partnering 20102017: Deal trends, players and financials [Report Updated: 01102017] Prices from USD $2995

Global Orphan Diseases Partnering 2010 to 2017 provides the full collection of Orphan Diseases disease deals signed between the world's pharmaceutical and biotechnology companies since 2010. Trends in...

H3 Biomedicine Granted Orphan Drug Designation of H3B-6527 for Treatment of Hepatocellular Carcinoma

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 ...

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Companies [57 Associated Companies listed on BioPortfolio]

Orphan Technologies

Orphan Technologies is dedicated to developing novel therapies to dramatically improve the lives of patients suffering from the rare disorder, homocystinuria and related diseases....

Rare Disease Communications®

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

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...

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...

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...

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 [110 Associated PubMed Articles listed on BioPortfolio]

Inter-species functional interactome of nuclear steroid receptors (R1).

Steroids exert their actions by binding to the glucocorticoid, mineralocorticoid, androgen, estrogen and progesterone classes of receptors. Despite an exponential increase in our knowledge of steroid ...

Discovery of new GPCR ligands to illuminate new biology.

Although a plurality of drugs target G-protein-coupled receptors (GPCRs), most have emerged from classical medicinal chemistry and pharmacology programs and resemble one another structurally and funct...

Structural prediction of two novel human atypical SLC transporters, MFSD4A and MFSD9, and their neuroanatomical distribution in mice.

Out of the 430 known solute carriers (SLC) in humans, 30% are still orphan transporters regarding structure, distribution or function. Approximately one third of all SLCs belong to the evolutionary co...

Mfsd2b is essential for the sphingosine-1-phosphate export in erythrocytes and platelets.

Sphingosine-1-phosphate (S1P), a potent signalling lipid secreted by red blood cells and platelets, plays numerous biologically significant roles. However, the identity of its long-sought exporter is ...

Demonstrating significant benefit of orphan medicines: analysis of 15 years of experience in Europe.

In the European Union demonstration of 'significant benefit' is mandatory if satisfactory methods exist for a disease targeted by a new orphan medicinal product. Significant benefit is required at the...

PLncPRO for prediction of long non-coding RNAs (lncRNAs) in plants and its application for discovery of abiotic stress-responsive lncRNAs in rice and chickpea.

Long non-coding RNAs (lncRNAs) make up a significant portion of non-coding RNAs and are involved in a variety of biological processes. Accurate identification/annotation of lncRNAs is the primary step...

Nr4a1 plays a crucial modulatory role in Th1/Th17 cell responses and CNS autoimmunity.

Nuclear receptor4 group A1 (Nr4a1), an orphan nuclear receptor, is down-regulated in peripheral blood mononuclear cells (MNCs) of individuals with multiple sclerosis (MS), and Nr4a1 deficiency results...

Establishment of primary mixed cell cultures from spontaneous canine mammary tumors: Characterization of classic and new cancer-associated molecules.

There are many factors which make canine cancer like cancer in humans. The occurrence of spontaneous mammary tumors in pet dogs, tumor genetics, molecular targets and exposure to the same environmenta...

Depot-Specific Response of Adipose Tissue to Diet-Induced Inflammation: The Retinoid-Related Orphan Receptor α (RORα) Involved?

Epididymal adipose tissue (EAT), a visceral fat depot, is more closely associated with metabolic dysfunction than inguinal adipose tissue (IAT), a subcutaneous depot. This study evaluated whether the ...

The Use of Social Media in Orphan Drug Development.

Social media has transformed how people interact with one another through the Internet, and it has the potential to do the same for orphan drug development. Currently, social media influences the orph...

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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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