Author Archives: remco

Importance rare disease patient advocacy recognized in scientific literature

The concept of patient advocacy organizations and its added-value in the rare disease area is certainly not new. However, a new trend may well be that advocacy initiatives are increasingly becoming the topic of a scientific publication. Many publications are … Continue reading

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Posted in patient role, Rare disease research | 4 Comments

RANKL cytokine: potential cure for rare bone disease

putting science into practice (1) The RANKL cytokine acts as the primary signal for bone removal, and as such plays a crucial role in bone homeostasis. In 2010, Amgen’s denosumab, a monoclonal antibody against RANKL was approved in the US … Continue reading

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“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

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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

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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

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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

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Posted in patient role, Rare disease research | 2 Comments

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

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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

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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

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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

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Posted in pharmaceutical innovation | 4 Comments