Orphan film disease

Orphan film disease

ViroPharma profiles orphan disease HAE in new film ViroPharma s new film, Swell, chronicles the trials of living with the rare disease hereditary angioedema, PRWeek reports (subscription required). The genetic disorder, which is potentially fatal, is…

US Conference Rare diseases orphan Products

US Conference Rare diseases orphan Products

NBIA Alliance members will present a poster on TIRCON and the NBIA Alliance at the European Conference on Rare Diseases & Orphan Products in Berlin. The conference is from May 8-10 and the poster will be presented on Friday, May 9. The poster…

Orphan diseases industry groups

Orphan diseases industry groups

Patient advocacy has always played a big role in the world of rare diseases. But as the industry has developed, it’s become clear that the patients really do need to be at the heart of the industry when it comes to orphan drugs. Patient involvement in…

Orphan diseases prevalence

Orphan diseases prevalence

Rare Diseases in numbers(3 MB) is a bibliographic study of the epidemiology of rare diseases initiated by EURORDIS in partnership with Orphanet. The study aims to evaluate the prevalence of a number of rare diseases in Europe and to document, for each…

Orphan diseases Congress

Orphan diseases Congress

Dr Geoffrey Guy is the founder of GW Pharmaceuticals and has served as Chairman since 1998. Dr Guy has over 30 years of experience in medical research and global drug development, most recently as Chairman and Chief Executive of Ethical Holdings plc (now…

Orphan diseases definition EMEA

Orphan diseases definition EMEA

Earlier this week, the European Medicines Agency (“EMEA”) announced that EMEA and FDA have adopted a common application form for sponsors seeking orphan drug designation in the European Union (“EU”) and the U.S. The move to a common application form was…

CAPS orphan disease

CAPS orphan disease

As with all very rare diseases, the clinical development program with canakinumab included a very limited number of patients for a necessarily brief period of follow-up; therefore, the post-approval period was deemed a critical phase in which to gather…

Orphan diseases venture capital

Orphan diseases venture capital

As high volume drugs become harder and more expensive to create and generics continue to put a squeeze on those margins, more companies are turning to less mainstream targets. Dr Sarah Houlton looks at the challenges and potential gains from orphan drugs…

Orphan lung disease definition

Orphan lung disease definition

Pulmonary vasculitis refers to inflammation of the small blood vessels in the lungs. It is usually part of a wider condition affecting the whole body, resulting in inflammation in the vessels within several organs. It can be treated with corticosteroids…

Orphan diseases companies

Orphan diseases companies

By Olga Oksman Trying to find the next hot space in pharmaceutical M&A is like shooting at a moving target. Or perhaps more aptly, a bobbing and weaving target. One area getting a lot of attention is the orphan drug space, industry bankers tell…

Orphan neuromuscular diseases

Orphan neuromuscular diseases

The multidisciplinary Center for Pediatric Neuromuscular and Orphan Diseases at the University of Florida is founded on the philosophy that integrated, interdisciplinary care is the most effective approach for patients with Neuromuscular and Orphan Diseases…

Orphan diseases UK

Orphan diseases UK

A rare disease is defined by the European Union as one that affects less than 5 in 10, of the general population. There are between 6, and 8, known rare diseases. Around five new rare diseases are described in medical literature each week. 1 in 17…

Orphan disease Cystic fibrosis

Orphan disease Cystic fibrosis

Cystic fibrosis (CF) is an inherited condition that causes mucus to build up and clog some of the organs in the body, particularly in the lungs and pancreas. When mucus clogs the lungs, it can make breathing very difficult. The thick mucus also causes…

Orphan diseases experience

Orphan diseases experience

Orphan Disease Marketing Orphan Disease Marketing REALITY Rx communication, a Global Healthcare Boutique, has a unique therapeutic focus for marketing products developed for orphan diseases and elevating awareness of these disorders. Our strategies and…

Orphan diseases review

Orphan diseases review

Deals Review of Lysosomal Storage Diseases and Other Orphan Indications Deals coverage on Cortellis Competitive Intelligence indicates an increasing trend in the number of deals that have been forged in the area of lysosomal storage diseases (LSDs) and…

Definition of a Rare disease

Definition of a Rare disease

Target of the lack of preparation of most doctors, forgotten by governments, dismissed by the farmaceutical industry and unknown by citizens, rare diseases cause suffering to those who have them and the people around them. More than an illness that will…

Orphan disease Philippines

Orphan disease Philippines

As in the past 5 years, The Philippine Society for Orphan Disorders, Inc. joins the global community of physicians patient organizations, and advocates in celebrating Rare Disease Day every last day of February. This year, Feb. 28 martks the 7th international…

Orphan disease incidence

Orphan disease incidence

Other Contributors: Harvard University-MIT Division of Health Sciences and Technology. Advisor: Jeff Behrens and Isaac Kohane. Department: Understanding the true incidence and prevalence of a disease has tremendous value for the biopharmaceutical industry…

Number of orphan diseases

Number of orphan diseases

Groft’s journey, which includes many major milestones, started on a personal note. Growing up in the 1950s, I had friends and neighbors who were stricken with diseases that had few or no effective treatments,Groft said. Some of the diseases of our time…

Orphan Annie disease

Orphan Annie disease

I was following a discussion on LinkedIn the other day about how much it costs to develop a NCE (new chemical entity/API). One of the contributors stated that, out of 100 NCEs, perhaps only one may ever get to the clinical trial stage. About the same…