France’s Patient Advocacy Power
France’s patient advocacy network is one of Europe’s most well-developed and formalised. Patient advocacy groups – including the three featured below – fill in research, care, and support gaps left…
France is considered among the leading rare disease markets worldwide, both scientifically and organisationally. The country has cultivated a fully-fledged supporting ecosystem for rare conditions that is today regarded as a benchmark internationally, and which few other countries have proven…
France’s patient advocacy network is one of Europe’s most well-developed and formalised. Patient advocacy groups – including the three featured below – fill in research, care, and support gaps left…
As rare disease regulation moves from the margins to the mainstream of global health policy, India faces both a challenge and an opportunity: how to align with international best practices…
More than 40 years ago in 1983, the Orphan Drug Act was passed in the US to support and incentivise the development of rare disease therapies. Despite the over 6,000…
The opportunity to continue growing the organisation, while driving performance and innovation, remains a strong source of motivation Sobi’s rapid growth in Germany and across DACH-NL is opening a new chapter for the rare disease specialist, as an expanding portfolio, new therapeutic areas and a growing organisation reshape the business. Wolfgang Walter discusses the strategy behind this momentum, the realities…
DACH should become a powerhouse for the Group Germany is emerging as a key growth and launch market for Ipsen, with the DACH region now ranking as the Group’s leading…
The rare disease landscape is becoming increasingly sophisticated, demanding not only scientific innovation but also new approaches to access, evidence generation and patient support. Recordati Rare Diseases has built its…
France has long been held up as Europe’s reference point for rare disease care – the first EU country with a national plan, a pioneer of coordinated expert networks, and…
Once regarded as a strategic afterthought by international biopharma, the Middle East has undergone a profound transformation in recent years, emerging as an early adopter of next-generation therapies for rare…
Patients and carers with lived experience of a disease have valuable insights to share with physicians and drug developers alike. This is especially true of rare diseases, defined in the…
When Nasha Fitter’s daughter, Amara, was diagnosed with the ultra-rare FOXG1 syndrome at just seven months old, the prognosis was stark: no speech, no independent movement, severe intellectual disability, and…
Long regarded as a clinical trials backwater, the Middle East has experienced a tremendous surge in clinical research over the past five years. This boom is fuelled by a global…
The US rare and ultra-rare disease market constitutes a cornerstone of the worldwide pharmaceutical industry, representing a full 52.3 percent of the total global market share in revenue for that…
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