Category: News

Fondazione Telethon announces commitment to make gene therapy for Wiskott-Aldrich Syndrome available to patients

Today, 28th of February, Fondazione Telethon (Italy) launched a Press Release to announce the agreement between Fondazione Telethon and Orchard Therapeutics, under which the foundation will work to make gene therapy for Wiskott-Aldrich syndrome available to patients. Read the Press Release here: https://www.telethon.it/en/stories-and-news/news/from-telethon-foundation/fondazione-telethon-announces-commitment-to-make-gene-therapy-for-wiskott-aldrich-syndrome-available-to-patients

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IRDiRC at 2024 WODC USA

As part of our commitment in advancing initiatives related to rare diseases, IRDiRC is a proud partner at the 2024 World Orphan Drug Congress USA. This will be a defining event for the rare disease and orphan drug space globally, bringing together top key opinion leaders from around the globe. The upcoming event will cover […]

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SANOFI highlights recent partnership to generate Real-World Evidence

The International Niemann-Pick Disease Registry (INPDR) announced a new collaboration with Sanofi, by giving controlled access to anonymized registry information via INPDR’s research “Gateway” platform. This collaboration will provide Sanofi with Real-World Evidence that will support evaluation and decision making on the use of olipudase alfa to manage Acid Sphingomyelinase Deficiency (ASMD). INPDR is the […]

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Celebrate the Rare Disease Day with CORD!

Join the Canadian Organization for Rare Disorders (CORD) in celebrating the most impactful Rare Disease Day! This year’s edition marks the Anniversary of the announcement of Canada’s first National Rare Disease strategy with an allocation of 1.5 billion dollars over 3 years. In addition, CORD will celebrate the launch of the first ever Rare Disease […]

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Rare Disease Day at Sanford Research

Rare Disease Day is celebrated on the last day of February every year and is a special day for rare disease patients, researchers, and advocates. There are over 300 million people worldwide living with a rare disease, and together across borders and the 10,000+ rare diseases, we work towards more equitable access to diagnosis, treatment, care, […]

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Join the Chinese Global Rare Diseases Research Symposium on May 23-25 in Shanghai!

IRDiRC is delighted to co-host, together with Hope for Rare Foundation and Fudan University, the Chinese Global Rare Diseases Research Symposium & The Second China Rare Disease Research and Translational Medicine Annual Conference on May 23-25 in Shanghai (China). The conference will last for 2.5 days with 21 parallel sessions and several satellite meetings. It […]

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Committee for Orphan Medicinal Products Plenary Meeting

The Committee for Orphan Medicinal Products (COMP) organized their Plenary Meeting last week (13-15 February), that was chaired by Regulatory Scientific Committee (RSC) Vice Chair, Violeta Stoyanova-Beninska, and Armando Magrelli. Among the topics discussed were the new applications for orphan medicinal product designation (including amendments of existing designations), requests for protocol assistance with significant benefit […]

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Rare Disease South Africa: get your #RarenessAwareness

Rare Disease Day is the globally-coordinated movement on rare diseases, working towards equity in social opportunity, healthcare, and access to diagnosis and therapies for people living with a rare disease. Since its creation in 2008, Rare Disease Day has played a critical part in building an international rare disease community that is multi-disease, global, and […]

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CORD Rare Disease Day Summit

The Canadian Organization for Rare Disorders (CORD) is very excited to be celebrating this once-in-four- years February 29th Rare Disease Day in Ottawa (Canada) on February 28-29, 2024. A panel, “Canadian Pathway to Rare Disease Drug Access”, is also scheduled on February 28 at 10:45 AM – 12:00 PM (local Ottawa time). This year, there […]

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