Topics

Orphan Technologies Company Profile

23:29 EDT 15th September 2019 | BioPortfolio

Orphan Technologies is dedicated to developing novel therapies to dramatically improve the lives of patients suffering from the rare disorder, homocystinuria and related diseases. OT-58, our lead drug development candidate, has been optimized as an enzyme replacement therapy for classical homocystinuria, a genetic disease characterized by debilitating cardiovascular, skeletal, neurologic, and ophthalmologic complications. OT-58 is designed to reduce homocysteine levels via a targeted mechanism of action and may have therapeutic applications in other diseases. For more information, please visit www.orphantechnologies.com


News Articles [640 Associated News Articles listed on BioPortfolio]

Orphan Technologies starts dosing in OT-58’s Phase I/II trial

Orphan Technologies has commenced dosing patients in a Phase I/II CBS-HCY-01 clinical trial of OT-58 for the treatment of classical...Read More... The post Orphan Technologies starts dosing in OT-58...

Palatin Gets Orphan Drug Designation For Melanocortin Agonist Peptide From FDA

WASHINGTON (dpa-AFX) - Palatin Technologies Inc. (PTN) said Thursday that the U.S. Food and Drug Administration granted orphan drug designation for PL-8177 for the treatment of non-infectious inte...

Pharmaceutical companies with the most orphan drugs

Orphan drug exclusivity is granted to drugs addressing rare ‘orphan diseases’ — those affecting fewer than 200,000 Americans. The protection granted to orphan drugs is quite strong. The FDA is...

The Rise of Orphan Drugs: A New Paradigm for the Future

In 1983, the United States Congress passed the Orphan Drugs Act designed to encourage the development of drugs for rare orphan diseases. The EU followed suit in early 2000 as well as a number of other...

The Orphan Drug Act Revisited

This Viewpoint reviews unintended consequences of the 1983 Orphan Drug Act, which incentivized development of drugs for rare diseases, including abbreviated approval processes for manufacturers and su...

Open Orphan PLC - integration on track one week on from IPO

Cathal Friel, chief executive of Open Orphan PLC (ORPH), caught up with Proactive London's Andrew Scott a week on from its re-admission to AIM. Friel reversed Open Orphan into Venn Life Sciences i...

The world’s top ten orphan drugs in oncology in 2018

The top orphan drugs in oncology in 2018: Ranking the ten biggest orphan drugs 10. Pomalyst/Imnovid (pomalidomide) 9. Alimta (pemetrexed)...Read More... The post The world’s top ten orphan drugs in ...

Mustang Bio Receives Orphan Drug Designation for MB-102

Mustang Bio (NASDAQ:MBIO) announced it has received orphan drug designation from the US Food and Drug Administration (FDA) For its MB-102 product to treat acute myeloid leukemia (AML). As quoted in th...

PubMed Articles [438 Associated PubMed Articles listed on BioPortfolio]

Modernising breeding for orphan crops: tools, methodologies, and beyond.

In spite of the limited investment in orphan crops, access to new technologies such as bioinformatics and low-cost genotyping opens new doors to modernise their breeding effectively. Innovation in pla...

The Impact of the Orphan Drug Act on FDA-Approved Therapies for Rare Skin Diseases and Skin-Related Cancers.

The Orphan Drug Act of 1983 (ODA) put in place a set of financial and marketing incentives to stimulate the development of drugs to treat rare diseases, and since its passage more than 600 orphan drug...

Analysing criteria for price and reimbursement of orphan drugs in Spain.

There are differences between countries regarding data requirements for orphan drug evaluation and it  is also unknown which criteria might determine the price and reimbursement decision. This stud...

The impact of orphan drug policies in treating rare diseases.

This paper is based on Shalini Weerasooriya's Masters dissertation in Public Health at the University of Sheffield. A literature review was conducted to understand the role that orphan drug policies h...

Role of healthcare databases and registries for surveillance of orphan drugs in the real-world setting: the Italian case study.

Orphan drugs are medicines intended to treat, prevent, or diagnose a rare and serious condition. The collection of pre-marketing safety and efficacy data for orphan drugs is very challenging for sever...

Clinical Trials [597 Associated Clinical Trials listed on BioPortfolio]

Safety and Feasibility of Normothermic Machine Perfusion to Rescue Orphan Livers

Orphan livers are organs that have been declined for clinical use by all centers due to their marginality. The current standard of care of liver preservation before transplant is cold stor...

Application of Monitoring and Intervention Technologies in Suboptimal Health Status

The purpose of this study is to use monitoring and intervention technologies in suboptimal health status in a general population in China and evaluate the effectiveness of these technologi...

Wheeze Detection in Adults During Dynamic Bronchial Situations Measured by PulmoTrack® 2010 With WIM-PC™ Technologies Recording

The PulmoTrack® 2010 with WIM-PC™ Technologies device, indicated for acoustic pulmonary function measurement that quantifies the presence of wheezing. This study was designed in order t...

Testing Latest Version of NidekRS3000Adv

The investigators would like to know if different imaging devices can improve the quality of images and visualization of imaged tissues. Also, the investigators would like to find out whet...

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...

Companies [1437 Associated Companies listed on BioPortfolio]

AOP Orphan Pharmaceuticals AG

AOP Orphan is a multinational company with headquarters in Vienna, Austria, focusing on clinical research, development and distribution of medicines for rare and complex diseases....

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....

Orphan Europe (UK) Ltd.

Orphan Europe was started with the objective of providing orphan drugs to patients afflicted by rare disorders.Over the years an information network has been established to increase the awareness abou...

Orphan Medical Incorporated

Orphan Medical Incorporated is a development stage company, was formed to acquire, develop and market products of high medical value intended to address inadequately treated or uncommon diseases of we...

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...

More Information about "Orphan Technologies" on BioPortfolio

We have published hundreds of Orphan Technologies news stories on BioPortfolio along with dozens of Orphan Technologies Clinical Trials and PubMed Articles about Orphan Technologies for you to read. In addition to the medical data, news and clinical trials, BioPortfolio also has a large collection of Orphan Technologies Companies in our database. You can also find out about relevant Orphan Technologies Drugs and Medications on this site too.

Quick Search

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...

Enzymes
Enzymes are proteins that catalyze (i.e., increase the rates of) chemical reactions. In enzymatic reactions, the molecules at the beginning of the process, called substrates, are converted into different molecules, called products. Almost all chemical re...


Corporate Database Quicklinks



Searches Linking to this Company Record