Orphan disease articles

Orphan disease articles

Wendy White shares details of an upcoming hackathon that will help to develop a screening tool that can alert primary physicians to the possibility of a genetic disorder and suggest a possible…
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Rare diseases and orphan Drugs Conference

Rare diseases and orphan Drugs Conference

In 2012, CBI is proud to present its 7th Annual Rare Disease and Orphan Drug Leadership Congress at a critical time for this community. Advocates of the 5, 800 rare diseases that have no…
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Orphan diseases Research UK

Orphan diseases Research UK

Being rare is becoming increasingly common. As patient groups such as Rare Disease UK rightly point out, collectively rare diseases affect roughly 1 in 17 people at some point in their life (approximating to around 3.5 million people in the UK). The research…

Orphan diseases Switzerland

Orphan diseases Switzerland

Dr. Rudolph Widmann established AOP Orphan in Vienna in 1996 in order to provide individualized treatments for patients suffering from Rare Diseases. AOP Orphan has especially focused in the areas of hematology and oncology, cardiology, pulmonology, neurology…

FDA orphan diseases database

FDA orphan diseases database

The concept is called “repurposing” — finding new ways to use products that have already been okayed by the FDA for some other use. The already-approved products in the Rare Disease Repurposing Database are unique in that they’ve also previously received…

Drugs diseases ultra orphan

Drugs diseases ultra orphan

Biotech companies developing and marketing orphan and ultra-orphan drugs have done exceptionally well in 2013 and in recent years. These include such names as: Biomarin (NASDAQ:BMRN), Alexion (NASDAQ:ALXN), NPS Pharma (NASDAQ:NPSP), Synageva (NASDAQ:GEVA)…

Orphan disease names

Orphan disease names

Distinguishable biosimilar names required to give rare disease patients optimum levels of care. In a letter to the US Food and Drug Administration, the National Organization for Rare disorders, has urged the body to take into condieration the impact of…

Orphan disease Penn

Orphan disease Penn

Welcome to the Center for Orphan Disease Research & Therapy at the University of Pennsylvania H. Lee Sweeney, Ph.D., William Maul Measey Professor of Physiology Director, Center for Orphan Disease Research and Therapy Orphan diseases represent…

NIH orphan diseases program

NIH orphan diseases program

This trans-NIH program has two main goals: To provide answers to patients with mysterious conditions that have long eluded diagnosis and To advance medical knowledge about rare and common diseases. How will the program define an undiagnosed disease? Any…

Rare diseases South Africa

Rare diseases South Africa

What began as a simple challenge amongst friends and family to raise awareness of Pompe Disease using the Momentum 94.7 Cycle Challenge as a platform, has evolved into charity initiative which seeks to help as many children and adults with rare conditions…

Rare Disorders or disease

Rare Disorders or disease

Statistics and Figures on Prevalence of Genetic and Rare Diseases Although rare and genetic diseases, and many times the symptoms, are uncommon to most doctors, rare diseases as a whole represent a large medical challenge. Combine this with the lack of…

Rare diseases and orphan drug registries

Rare diseases and orphan drug registries

The International Workshop “Rare Disease and Orphan Drug Registries” is organized in the framework of the EPIRARE project (“European Platform for Rare Disease Registries”, co-funded by EU Commission, DG SANCO). It is open to scientists, clinicians, patients’…

Rare diseases European Union

Rare diseases European Union

This section on health in the EU has been created as a trustworthy gateway to a wide range of information and data on health-related issues and activities at both European, national and international level. The content is produced by the European Commission…

Orphan diseases ALS

Orphan diseases ALS

Why Orphan Diseases Should Be a Jewish Priority (Guest Author: Avichai Kremer of Prize4Life) When I first learned about Prize4Life, I was unsure if ALS (also known as Lou Gehrig’s disease) was a Jewish issue. But now I am convinced. Read more to find…

Orphan infectious diseases

Orphan infectious diseases

Medical Resources :: Common Diseases :: Infectious Diseases & Parasites :: Tuberculosis as published in the International Primer The epidemiology of Tuberculosis abroad is well-understood. The high prevalence countries are Mexico, the Philippines…

Orphan disease Treatment Institute

Orphan disease Treatment Institute

Orphan Disease Overview : One of the institute s priorities is to develop diseased and patient specific stem cells for drug discovery initiatives to find treatments for orphan (rare) diseases. The institute is collaborating with Cellular Engineering Technologies…

Ultra orphan diseases definition

Ultra orphan diseases definition

Lysosomal Storage Disease: an Example of a Lucrative Ultra-Orphan Market The pharmaceutical industry has shown an increasing interest in developing drugs for rare, orphan diseases. Amongst the orphan diseases is a subgroup of extremely rare disorders…

FDA orphan disease granted

FDA orphan disease granted

In order to access this content you need to be logged into the site and have an active subscription or trial subscription. Please login, take a free trial or subscribe in order to continue reading. A trial subscription will give you access to the latest…

Orphan diseases definition European

Orphan diseases definition European

Definition of centre of reference in European countries There is no common definition of what a centre of reference is among those Member States which have established such centres. Even the definition of a rare disease varies between countries with official…

Orphan disease multiple sclerosis

Orphan disease multiple sclerosis

Multiple sclerosis (MS) is a disorder that affects the central nervous system (CNS), specifically the brain and spinal cord. A fatty tissue called myelin surrounds and protects the nerve fibers of the CNS. Myelin insulates the nerves and allows them to…

What does orphan disease means?

What does orphan disease means?

Frederick S. Kaplan, MD 1, 2, 4 Robert J. Pignolo, MD, PhD 1, 2, 4 Eileen M. Shore, PhD 1, 3, 4 (From The Departments of 1Orthopaedic Surgery, 2Medicine and 3Genetics and 4The Center for Research in FOP and Related Disorders, The Perelman School of Medicine…

Orphan receptor disease

Orphan receptor disease

Scientists at the Scripps Research Institute have reported on compounds that are able to suppress severity and disease progression in animal models of multiple sclerosis. The compounds, exemplified by SR1001, act by selectively suppressing a subset of…

Rare diseases and orphan drug Act

Rare diseases and orphan drug Act

Back Story Of The Orphan Drug Act Ronald Reagan is remembered for many things, but few would associate his presidency with the subject of medicinals. Yet it was Reagan who signed into law the two acts that have, during the past twenty-five years, had…

Orphan disease Genzyme

Orphan disease Genzyme

CAMBRIDGE, Mass.-(BUSINESS WIRE)-Genzyme, a Sanofi company (EURONEXT: SAN and NYSE: SNY), today announced its support of International Rare Disease Day with a series of initiatives meant to call attention to rare diseases as an important public health…