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  • Latest rare diseases news stories and updates. What is happening today? All recent rare diseases news and developments. Live coverage and timeline of rare diseases. All you need to know about rare diseases.
Hidden Sunbed Danger Warning
New Analyses Advance Understanding of Long-Term Survival Outcomes with Mogamulizumab in Mycosis Fungoides and Sézary Syndrome
World Health Assembly endorses resolution on precision medicine
Research Project Promoting Patient-Specific Nucleic Acid Drug Development for Rare and Intractable Diseases (N-of-1+ Drug Development) Selected by AMED
New spin-out to make life-saving medicines more affordable
Eneboparatide normalised serum calcium and achieved independence from active vitamin D and oral calcium supplements in 31.1% of adults with hypoparathyroidism at…
UQ Discovery Boosts Leading Gene-editing Tool
Update on FDA Advisory Committee vote on camizestrant in combination with a CDK4/6 inhibitor for advanced HR-positive breast cancer
Breztri approved in the US for asthma as first and only triple therapy for patients 12 years of age and older
Saphnelo approved in the US for subcutaneous self-administration as a new autoinjector for the treatment of systemic lupus erythematosus
Tozorakimab met primary endpoint in Phase III MIRANDA trial in patients with COPD
Alexion data at 2026 AAN Annual Meeting reflects industry-leading portfolio and commitment to enhancing care across rare diseases
Cheaper Medicines For People Fighting Cancer And Rare Diseases
Common Genetic Cause Of Severe Epilepsy Revealed
Precision medicine helps more patients receive a genetic diagnosis
Tozorakimab met primary endpoint in both OBERON and TITANIA Phase III trials in patients with COPD
Erdinc Sezgin: “The real outcome of KIRI is the next generation of researchers”
New infrastructure for mRNA production and lipid nanoparticle formulation
A new paradigm for medical AI: why disagreement between models may be more valuable than consensus
Study reveals worrying extent of imprecise gene and gene mutation naming
Positive and clinically meaningful results from the Phase III KALOS and LOGOS trials for Breztri in patients with uncontrolled asthma published in…
Tiny ‘mini-me’ organs grown from children’s cells transforming cystic fibrosis care
Update on US regulatory review of Saphnelo subcutaneous administration in systemic lupus erythematosus
AstraZeneca begins trading on the New York Stock Exchange
AstraZeneca enhances its weight management portfolio through collaboration agreement with CSPC Pharmaceuticals
Electronic Informed Consent in Research on Rare Diseases
$120 million for research to improve prevention, diagnosis, treatment and care
AstraZeneca to complete direct listing of ordinary shares and all US debt securities on the New York Stock Exchange
AstraZeneca appoints Joris Silon as Head of Investor Relations
Saphnelo self-administration TULIP-SC trial demonstrates statistically significant and clinically meaningful reduction in systemic lupus erythematosus disease activity
World leaders adopt a historic global declaration on noncommunicable diseases and mental health
NSW Government urged to develop rare disease strategy
Eight researchers at MMK receive research funding from the Swedish Research Council
New Life Saving Medicine Free For Rare Disease
Monash Scientists Uncover Genetic Code That Could Unlock Game-changing MRNA Disease Therapies
Focused Sound Energy Holds Promise For Treating Cancer, Alzheimer’s And Other Diseases
Tilen Trselic Awarded 2025 Cilla Weigelt Scholarship
Newborn Genomic Screening Enables More Lifesaving Diagnoses
New Care Centre To Change Lives Of Children With Rare Diseases
World leaders show strong support for political declaration on noncommunicable diseases and mental health
NIH awards more than $15 million to fund two rare diseases consortia
Purple Lights For Global ITP Awareness Week 2025
KFSHRC to Participate in C3 Davos of Healthcare(TM) Japan Summit
How Mutations In Bodily Tissues Affect Ageing
New route into cells could make gene therapies safer
Appendix cancer rising among younger generation – new study
WHO Director-General: Member States reaffirm commitment to WHO and global health at historic World Health Assembly
Seventy-eighth World Health Assembly concludes: historic outcomes, consequential highlights

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