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Latest rare diseases News

  • 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.
Curious Kids: how is eye colour made? And why are they different colours?
More rare conditions considered for newborn bloodspot screening and new treatment option for spinal muscular atrophy
Losing their tails provided our ape ancestors with an evolutionary advantage – but we’re still paying the price
Acquisition Of Gracell Completed
Acquisition of Icosavax Completed
Tagrisso demonstrated overwhelming efficacy benefit for patients with unresectable, Stage III EGFR-mutated lung cancer in LAURA Phase III trial
Game changing gene therapies focus of £14m research hub
New Cellular Lab To Back Victorian Blood Cancer Research
AstraZeneca to acquire Gracell, furthering cell therapy ambition across oncology and autoimmune diseases
Research aims to transform haemophilia treatment
Centenary researchers awarded more than $2m in Ideas Grants
Aegros Releases Top-Line Interim Clinical Trial Results of its COVID-19 Hyperimmune
Neurodegeneration in myelin disease: no myelin is better than bad myelin
Unlocking power of genomics with $66 million for research
Update on PACIFIC-2 Phase III trial of Imfinzi concurrently administered with platinum-based chemoradiotherapy in unresectable, Stage III non-small cell lung cancer
AstraZeneca announces collaboration and investment agreement with Cellectis to accelerate cell therapy and genomic medicine ambitions
$147.5 million for research into rare illnesses, multidisciplinary primary care, cardiovascular disease and diabetes
US Food and Drug Administration accepts for review AstraZeneca’s Supplemental Biologics License Application for self-administration of FluMist Quadrivalent
Shortage in regional nuclear medicine workforce attracts new financial support
Professor Maher Gandhi: Life as clinician-scientist
Tagrisso plus chemotherapy granted Priority Review in the US for patients with EGFR-mutated advanced lung cancer
Nation-First Stem Cell Trial Offers Hope To Young Australians
AstraZeneca awards funding to eight innovative research projects from early-career scientists around the world in second R&D Postdoctoral Challenge
US FDA grants Orphan Drug Designation for Noxopharm’s CRO-67
AstraZeneca research reveals unique associations between rare changes in genes and plasma proteins that could improve drug discovery with data from over…
Forxiga met primary endpoint in T2NOW Phase III trial, one of the largest paediatric type 2 diabetes studies performed to date
AstraZeneca settles Nexium and Prilosec product liability litigations
Alexion completes purchase and licence agreement for early-stage rare disease gene therapy portfolio from Pfizer
Syndesis Health and InSyBio Partner in Predictive Modelling and Biomarker Discovery
RACGP: General practice care crucial in helping patients with rare cancers
Fasenra met the primary endpoint in the MANDARA Phase III trial in eosinophilic granulomatosis with polyangiitis (EGPA)
Tagrisso plus chemotherapy extended median progression-free survival by nearly 9 months in EGFR-mutated advanced lung cancer in FLAURA2 Phase III trial
Calquence approved in China for chronic lymphocytic leukaemia
Forxiga approved in China to reduce the risk of cardiovascular death and hospitalisation in adult patients with symptomatic chronic heart failure
New ‘Long-term Insights briefing’ to examine role of technology in people’s health
Alexion, AstraZeneca Rare Disease, enters agreement with Pfizer to acquire a portfolio of preclinical rare disease gene therapies
AusBiotech launches National Cell and Gene Manufacturing Blueprint
Industry-led cell and gene manufacturing strategy to develop Australia as APAC hub launched
DHX9 variations underly wide spectrum of human neurodevelopmental disorders
Variations in gene DHX9 underly wide spectrum of human neurodevelopmental disorders and neuropathy
Restoring Lost Sense of Smell
Precision medicine for Indigenous communities
Solving rare disease mysteries. And protecting privacy
Ending postcode lottery in newborn health screening
Capivasertib in combination with Faslodex granted Priority Review in the US for patients with advanced HR-positive breast cancer
AstraZeneca announces agreement with Quell Therapeutics to develop, manufacture and commercialise engineered T-regulatory cell therapies for autoimmune diseases
Update on brazikumab development programme
Helping patients navigate rare diagnosis

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