Orphan disease exclusivity

Orphan disease exclusivity

There’s a first time for everything! And on August 8th, 2012, FDA decided, for the first time since the enactment of the Orphan Drug Act, to rescind a period of 7-year orphan drug exclusivity. The decision came in the form of a response granting in part…

Rare disease that causes blindness

Rare disease that causes blindness

However, it does not come cheap and is only available to private patients at a cost of 50, . Training to use the gadget also costs about 10, . Second Sight, the company which makes the technology, hopes that it will become available on the NHS. The implants…

Orphanet Journal of Rare diseases

Orphanet Journal of Rare diseases

Orphanet Journal of Rare Diseases is an open access, online journal that encompasses all aspects of rare diseases and orphan drugs. The journal publishes high-quality reviews on specific rare diseases. In addition, the journal may consider articles on…

Orphan diseases in USA

Orphan diseases in USA

The FDA Office of Orphan Products Development (OOPD) mission is to advance the evaluation and development of products (drugs, biologics, devices, or medical foods) that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions…

Orphan disease cancer

Orphan disease cancer

An important trial reported in this issue of Annals of Oncology builds upon this foundation and on the promising activity of paclitaxel in phase II trials. The randomised comparison of the standard three-weekly doxorubicin (60mg/m2) plus cisplatin (50mg/m2)…

Orphan diseases Geneva

Orphan diseases Geneva

It seems that there is an acceleration of interest in crowdfunding in the rare disease and orphan drug space. Rare Connect, an online community that connects rare disease patients globally, organizes a webinar this past October, to introduce how crowdfunding…

Rare cartilage disease

Rare cartilage disease

Learning about a Rare Disease (Relapsing Polychondritis) by Joe Rivas, Denton, TX Mary Sparkman with husband Steve and son Steve Jr. celebrating Christmas. Mary Sparkman at home. As a Rehabilitation Counselor and a promoter of health care awareness, I…

Ophthalmology orphan diseases

Ophthalmology orphan diseases

MANF continues to perform on so many different levels for Amarantus. While the recent focus has been on the Company’s nearer-term product candidate, it is critically important to remember the vast potential of our program. What I find most remarkable…

Rare diseases and orphan Drugs

Rare diseases and orphan Drugs

With the increase in pharmaceutical companies developing orphan drugs, there is a need for tools for rare disease patients and healthcare professionals to provide the capability to find clinical trials appropriate for their disease, geographic location…

Orphan disease symptoms

Orphan disease symptoms

Rare diseases are diseases which affect a small number of people compared to the general population and specific issues are raised in relation to their rarity. In Europe, a disease is considered to be rare when it affects 1 person per 2. A disease can…

Rare disease cant gain weight

Rare disease cant gain weight

A 12-year-old Texas girl with a rare weight-gain disorder has successfully raised more than $55, to pay for life-saving gastric bypass surgery. In 2011, Alexis Shapiro was diagnosed with a rare brain tumor and underwent surgery to remove the mass, according…

Orphan diseases European

Orphan diseases European

A disease or disorder is defined as rare in the EU when it affects less than five in every 10 citizens. Yet, because there are so many different rare diseases – between 6 and 8 – taken together they affect a significant share of the population. Between…

Orphan diseases and pediatric studies FDA

Orphan diseases and pediatric studies FDA

The Food and Drug Administration (FDA) recently released its FDASIA-mandated report and strategic plan on ways to encourage and accelerate the development of therapies for pediatric rare diseases. This plan builds on the FDA public meeting held in January…

Orphan disease Lab UCLA

Orphan disease Lab UCLA

Thomas Drake, MD Integrating genetics and gene expression to study complex disease pathogenesis Naturally occurring genetic variation among individuals affects many if not most complex biologic processes to some measurable degree. This variation can be…

Orphan diseases market

Orphan diseases market

Right now drugs for rare disorders are the sweet spot of the biotechnology industry. Treatments for illnesses that affect only hundreds of patients can cost $200, or more and have built Genzyme into one of biotech’s biggest companies and Alexion and…

EULAR orphan disease Programme

EULAR orphan disease Programme

The EULAR Orphan Disease Programme (ODP) on systemic sclerosis In 2007, EULAR defined its strategic objectives for the period 2008-2012. As part of this strategy, the Executive Committee decided to launch a 4-year orphan disease programme focusing on…

Orphan Autoimmune diseases

Orphan Autoimmune diseases

Idera’s business strategy is focused on developing targeted therapies to treat rare and severe autoimmune diseases with high unmet medical need. In autoimmune diseases, overactivation of Toll-like receptors (TLRs) by tissue and organ damage is implicated…

Orphan diseases and its management

Orphan diseases and its management

The web survey focused on the current management of 9 orphan agents selected to achieve a mix of oncology and nononcology agents, orphan and ultra-orphan drugs, various modes of administration, existence of treatment alternatives, and multiple indications…

Diseases in orphan

Diseases in orphan

Peter Saltonstall Rare Diseases: Orphans in Danger For rare diseases and orphan products, the future looks promising, but as Peter Saltonstall finds, significant research, educational and policy challenges remain. A view from one of the patient community…

Orphan diseases interleukin

Orphan diseases interleukin

Nearly 2 centuries have passed since Lannec wrote the first clinical description of a patient with bronquiectasis, 1 more than 80 years since bronchography became the standard diagnostic method, and more than half a century since Reid used histological…

Orphan disease liver

Orphan disease liver

While surged earlier this year and has surged this week after agreed to purchase hepatitis stock at a 239% premium, it seems the herd has realized that small cap is also a liver disease stock as shares suddenly surged 56.69% on no apparent company news…

Rare disease roadblocks

Rare disease roadblocks

About The EveryLife Foundation for Rare Diseases is dedicated to accelerating biotech innovation for rare disease treatments through science-driven public policy. We can do more with the science we already have and bring life-saving treatments to millions…

Orphan drug designation Fast Track

Orphan drug designation Fast Track

MONTREAL--Angiochem announced today that the Food & Drug Association (FDA) has granted both orphan drug and fast track designation to ANG1005 a novel paclitaxel-peptide drug conjugate leveraging the low density lipoprotein receptor-related protein…

Orphan diseases Treatment Institute Company Ltd

Orphan diseases Treatment Institute Company Ltd

Company name: DAIICHI SANKYO COMPANY, LIMITED Representative: Joji Nakayama, President and CEO (Code no.: 4568, First Section of Tokyo, Osaka and Nagoya Stock Exchanges) Please address inquiries to Noriaki Ishida, Corporate Officer, Vice President, Corporate…