top of page
All Posts


It is time to shift the paradigm on rare diseases
It’s time to make rare diseases a global health priority and ensure that people living with rare diseases are not left behind.
Alexandra Heumber Perry—Global
4 min read


Can high-cost gene therapies become affordable for all?
As new gene therapies offer hope for rare diseases, health systems work to balance innovation with sustainability.
Jo Waters—Global
9 min read


AI transforming rare diseases diagnosis and care
AI is revolutionising rare disease research by bridging data gaps and enabling faster diagnoses and more effective treatments.
Maurizio Arseni—Global
8 min read


Challenges and opportunities for genomics in Africa
Genomic testing in Africa would benefit from more awareness and a national registry of patients living with rare diseases across Africa.
Trudy Nyakambangwe—Zimbabwe
6 min read


Uganda takes action to ease sickle cell disease burden
In Uganda, sickle cell disease burdens families with social isolation and high costs. Growing awareness offers hope.
Zarina Geloo—Zambia
8 min read


Designing a collaborative care system beyond caretakers
Rare disease patient management must include providing both long-term support for patients and peace of mind for families.
Prasanna Shirol—India
4 min read


The forgotten social and emotional toll of rare diseases
People with rare diseases face stigma, isolation, and neglect. Addressing social and emotional impacts is crucial for better support.
Becky McCall — Global
7 min read


South Africa’s fight for fair rare diseases healthcare
In South Africa, rare disease patients struggle for diagnosis and care. While resources are limited, advocacy groups push for reforms.
Hannane Ferdjani—South Africa
8 min read


Finding strength through innovation and inclusion
Assistive technology and inclusion in the workplace can drive independence for people with rare diseases.
Christhyl Ceriche—Chile
5 min read


How lived experience powers rare disease advocacy
Innovative collaborations are helping rare disease patient groups campaign for change and training medical students to be advocates.
Danny Buckland—Global
7 min read


Bringing rare eye diseases into focus
Rare eye diseases are often overlooked. Integrating rare disease and eye health initiatives ensures access to universal care.
Junu Shrestha—Global
4 min read


The new frontier of genomic testing for rare diseases
Genomic technology is revolutionising the diagnosis and treatment of rare diseases. The next step is ensuring equitable access.
Paul Adepoju — Nigeria
8 min read


Advancing rare disease advocacy in Kenya
Significant strides have been made to bring advocacy and engagement on rare diseases from Kenya to the global stage.
Christine Mutena—Kenya
4 min read
bottom of page