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Latest rare diseases News | Page 2

  • 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.
Update on brazikumab development programme
Helping patients navigate rare diagnosis
AI drug discovery will power collaboration with Pharos Therapeutics
New funding to advance gene therapies
Critical technologies securing Australia’s future
Tagrisso plus chemotherapy demonstrated strong improvement in progression-free survival for patients with EGFR-mutated advanced lung cancer in FLAURA2 Phase III trial
New report calls on national cell and gene therapy plan
Children with complex needs benefit from charity funds
PRIME MINISTER BREAKS ELECTION COMMITMENT TO AUSTRALIAN BABIES
Update to contractual arrangements between AstraZeneca, Swedish Orphan Biovitrum AB and Sanofi
Social solution – rare ovarian cancer insights
License agreement with KYM Biosciences for CMG901, a Claudin-18.2 antibody drug conjugate, completed
Eplontersen demonstrated sustained benefit in Phase III trial for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) through 66 weeks
TGA Product Information safety updates 27 March
Calquence granted first regulatory approval in China for adults with previously treated mantle cell lymphoma
CSL reports positive results from pivotal late-stage trial of first-in-class garadacimab
Big impact of rare disease research
Genomics key to First Nations health equality
CSL Closing of Arcturus Therapeutics Agreement
More medicines for more New Zealanders
AstraZeneca showcases strength of haematology portfolio and pipeline across multiple hard-to-treat conditions
AstraZeneca announces sale of its West Chester, Ohio manufacturing site
AstraZeneca to acquire Neogene Therapeutics, accelerating ambition in Oncology cell therapy
U.S. Food and Drug Administration approves CSL’s HEMGENIX , first gene therapy for hemophilia B
UN expert calls for lifting of long-lasting unilateral sanctions ‘suffocating’ Syrian people
Major cerebral palsy gene revealed
Gene technologies are giving hope to patients with deadly rare diseases
Gene technologies are giving hope to patients with deadliest rare diseases
AstraZeneca funds R&D Postdoctoral Challenge finalists
Vaxzevria receives full Marketing Authorisation in EU for prevention of Covid
Camizestrant significantly improved progression-free survival in advanced ER-positive breast cancer
Capivasertib Phase III trial met primary endpoints
Update on MESSINA Phase III trial for Fasenra in eosinophilic esophagitis
Alexion aims to advance NMOSD treatment landscape with exceptional Ultomiris efficacy data at ECTRIMS 2022
Strengthening healthcare systems to meet patients’ need for plasma-derived therapies
New data show Farxiga significantly lowers risk of cardiovascular death in patients with heart failure
Drug shows promise for motor neuron disease
Acquisition of TeneoTwo for its clinical-stage T-cell engager completed
Calquence tablet formulation approved in US across current indications
Michel Demaré to succeed Leif Johansson as Non-Executive Chair of Board
Labor Will End Newborn Health Screening Lottery

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