Rare Diseases Q3 2024

Momentum grows towards a WHA Resolution on rare diseases

Equality in rare: building a better future for people with rare diseases

Fighting for healthcare equity: rare disease medications in emerging markets

How light has become a constant threat to patients with a painful rare disease

Sharing the health decision lightens your load

Connect with others and share your authentic rare disease patient story

European legislation must be revised to boost innovation in rare disease

This article has been developed and funded by Chiesi Group Rare Diseases. Inflation and rising public debt are creating barriers for the rare disease community to access the latest therapies. Public healthcare budgets are being reduced, making access to new treatments difficult while stricter evidence requirements further limit availability across Europe. The lack of treatment … Continued

Ways to champion people-powered communications in rare diseases

Woman Speaking At Support Group Meeting For Mental Health Or Dependency Issues In Community Space

How women are revolutionising rare disease

How to prove orphan drug cost-effectiveness and safety to secure market access

Rare Diseases 2025

Protected: Gain insights from the largest orphan drug and rare disease event globally

People, medical team and laptop for collaboration, advice and discussion for surgery or patient records. Doctor, technology and professional opinion for help, results and health in clinic or hospital

Rare Diseases 2025

Protected: Clinical clues pointing to rare disease

Protected: The rare disease community — why it’s more than you can imagine

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Protected: Why patient voices can help unlock rare disease treatment breakthroughs

Protected: Rare voices, bold advances: how MPN patients are helping shape medicine

Genomic analysis visualization. Dna genomes sequencing, deoxyribonucleic acid genetic map and genome sequence analyse vector concept

Rare Diseases 2025

Protected: Investigating genomic newborn screening for rare conditions

Mom, nurse and black child patient in hospital for recovery of surgery for congenital heart disease. Checkup, sick and cardiology medical worker with happy mother and kid for postoperative care

Protected: A brighter future for children with cancer

Rare Diseases 2025

Protected: Rare diseases and health equity: shifting the paradigm through global action

Therapy, happy women have a conversation on sofa and speaking or talking with a psychologist. Mental health or communication, support or consulting and people on couch have a discussion together

Rare Diseases 2025

Protected: Opportunities to improve the lives of those with rare conditions

There is no excerpt because this is a protected post.

Rare Diseases 2025

Protected: More than you can imagine: rare diseases in focus

This highly detailed image presents orange glowing bacteria floating against a dark background, emphasizing microbial life and its constant presence in our surroundings

Rare Diseases 2025

Protected: From understanding to impact: driving academic research to deliver rare therapies

Simplifying development of next-generation rare disease therapies

Help, support and medical with nurse and old man for retirement, rehabilitation or healing. Empathy, physical therapy and healthcare with patient and black woman in nursing home for caregiver service

Rare Diseases Q3 2024

How the UK can address sickle cell disease care inequalities and funding shortages

Rare Diseases Q3 2024

Free genomic sequencing offers hope for children living with a rare disease