This Rare Disease Day, Elsevier’s Scopus research and citation database analyses articles from millions of researchers, institutions and companies around the world to present data that suggests rare disease research has skyrocketed over the last ten years, but is starting to level off. According to the data, there was a… Read More
Rare Disease Day is an observance held on the last day of February annually to raise awareness for rare diseases and improve access to treatment and medical representation for individuals with rare diseases and their families. To mark Rare Disease Day 2025, DDW gathered insight from a range of industry… Read More
For Rare Disease Day 2024, Diana Spencer provides a round-up of the latest breakthroughs in the treatment of rare disease. Study reveals therapeutic potential of mRNA in rare diseases Researchers have used messenger RNA (mRNA) to create an effective therapy for a rare… Read More
By Lucy Radley, Head of EU Regulatory Development, ProPharma. A rare disease is defined as a condition which affects less than one in 2000 people. Although individually they are very rare, collectively they are common with one in 17 people (around 3.5 million) in the UK being affected by a… Read More
A new strategic guide, intended to help manufacturers bring rare disease products to market, has been launched to mark Rare Disease Day (February 28). In the guide, Terebellum (a subsidiary of AscellaHealth) shares tips on how industry stakeholders can create an effective market access strategy, giving their treatment the best chance… Read More
Rare diseases are often overlooked for funding and research, but with various incentives and priority review programmes now in place, many companies are beginning to explore these unusual but often devastating conditions. This Rare Disease Day we provide an overview of the progress that has been made in… Read More
Dr Asma Patel, Vice President of Integrated Services for Early Development at Quotient Sciences, reflects on the biggest barriers to rare disease drug development and how companies can overcome these. Despite improvements in regulatory support and commercial rewards, difficulties remain for drug developers in bringing rare disease drugs to market. Read More
mRNA technology helped us to vaccinate the world against SARS-CoV-2. As we turn our focus to new potential applications and disease areas for the platform, scientists and companies must consider the potential life-saving impact on rare, inherited diseases, says Archana Gupta, PhD, staff scientist in genetic sciences at Thermo Fisher… Read More
