November Is Lung Cancer Awareness Month
Did you know that lung cancer is one of the most common types of cancer worldwide? It is also the leading cause of cancer-related deaths...
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in the
press
- Oct 13
- 2 min
Celebrating Liesel…
Today is Phelan-McDermid day and we are celebrating Liesel . 5 year old Liesel was born in Pretoria, with Phelan-McDermid Syndrome, and...
- Oct 5
- 6 min
Switching and Monitoring Best Practice for Gaucher Disease
A consensus document Prepared by: International Working Group on Gaucher Disease (IWGGD) and International Gaucher Alliance (IGA) Issued...
- Oct 4
- 2 min
Living with Gaucher disease as a toddler in South Africa
Two-year-old Eden Green was admitted to Red Cross Children’s Hospital in July 2020, where she was found to have an enlarged liver and...
- Sep 22
- 3 min
Call on United Nations Member States to make Universal Health Coverage a Reality for Rare Diseases.
21 September 2023, New York – A high-level meeting at the United Nations Headquarters, UN Member States gathered to accelerate progress...
- Sep 19
- 4 min
Orchard Therapeutics Announces Acceptance of Biologics License Application for OTL-200 in MLD.
Hope is on the horizon for patients impacted by metachromatic leukodystrophy.
- Sep 6
- 3 min
Intellectual Property in Healthcare: the Patient Perspective
Intellectual Property in healthcare -what does it mean for patients?
- Aug 18
- 1 min
Statement of Intent of The Rare Diseases Working Group
The Rare Disease Access Initiative (RDAI), outlined in our Statement of Intent addresses the pressing challenges surrounding rare disease...
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