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Author Archives: remco
“Made in China” orphan drug may soon be reality
Napoleon Bonaparte once said of China, “Let her sleep, for when she wakes, she will shake the world.” When it comes to rare diseases China has awoken and belongs to the top-10 countries based on scientific output. China doesn’t just … Continue reading
What you always wanted to know about Orphan Drug development……
This week a wonderful overview of orphan drugs (OD) by Joppi, Bertele and Garattini was published in the October issue of the European Journal of Clinical Pharmacology. The overview covers in depth preclinical and clinical information for each OD approved in … Continue reading
Posted in pharmaceutical innovation
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The Wellcome Trust Pathfinder Award scheme: bridging the gap between R and D
The Wellcome Trust Pathfinder Award scheme represents an important new funding opportunity for early stage R&D projects in the area of rare diseases. Review of the eligibility criteria raises the question whether the Trust is fully aware of the central role that Small … Continue reading
Rare disease charities go beyond pump-priming research
The Myrovlytis Trust, a UK-based charity, is making a difference for patients suffering from Birt-Hogg-Dubé syndrome. It does so in many different ways and is accompanied by many more charities. Since its foundation in 2007 the Myrovlytis Trust has been … Continue reading
Posted in patient role, Rare disease research
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Ireland may be green, but its way of funding rare disease research is certainly not
In Europe we are in the middle of a huge endeavour: the establishment and implementation of strategies to combat rare diseases. The endeavour is not without challenges, but we will succeed, because the impact on the lives of rare disease … Continue reading
Posted in patient role, Rare disease research
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China has joined the fight against rare disorders
The occurence of rare disorders doesn’t stop at the US, Japan or EU border, but affect people all over the world. To be most effective, countries across the globe should join the fight against rare disorders. I provide preliminary data … Continue reading
Posted in Rare disease research
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Adult autologous stem cells: practice of medicine or drug product ?
Telemedicine, social media, mobile medical Apps, stem cells……. just a few examples of innovations that are quickly becoming an integral part of modern (rare disease) health care. At the same time these innovations in medicine seriously challenge our current regulatory framework. On 30 April 2012, … Continue reading
Posted in pharmaceutical innovation
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Estimating rare disease prevalence: a tough nut to crack ?
How can one retrieve reliable and acceptable data on rare disease prevalence and do specific resources exist that can be consulted ? That was an inquiry I recently received. Having received similar questions in the past, I wondered if orphan drug … Continue reading
Posted in pharmaceutical innovation
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To cure is good, to care is ……..
Added-value of social software platforms Developing therapies for rare disorders, so-called orphan drugs, is becoming increasingly popular within the pharmaceutical sector. However, developing a drug takes a long time: around 10-15 years. Moreover, for some rare diseases developing a curative … Continue reading
Posted in Care
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Patient-initiated research foundations: A source of funding, but more importantly a continuous source of inspiration
Some patient-initiated research foundations have been around for quite some time and have grown in considerable funds, others have just been founded and are still small. An intriguing question is what is their combined impact on rare disease research and orphan … Continue reading
Posted in patient role
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