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Rare Disease
Sio Gene Therapies Touts Positive Safety and Biomarker Data for GM1 Gangliosidosis Candidate
2021-10-22
FDA Greenlights First and Only Treatment for Cholestatic Pruritus with ALGS
2021-10-04
Merck Acquires Acceleron for $11.5 Billion, Gaining Access to a Phase 3 Cardiovascular Asset
2021-09-30
AstraZeneca Acquires Caelum in $500m Deal to Strengthen Rare Diseases Division
2021-09-30
Cancer Fears Puts BioMarin’s Phearless Study on Hold
2021-09-07
BeiGene’s Brukinsa Bags Second FDA Approval, to Rival Imbruvica in Treating a Rare Cancer
2021-09-06
Serious Adverse Event in Study Participant Marks Latest Roadblock for Astellas’ Gene Therapy Trial
2021-09-03
Merck Fortifies Oncology Pipeline with FDA Win for HIF-2α Inhibitor, Welireg
2021-08-17
New Gene Therapy Offers Hope to Reverse Rare Genetic Disorder in Children
2021-07-16
Venglustat’s Phase 2/3 Trial Failure Pushes Sanofi to Drop Kidney Disease Program
2021-06-02
FDA Rejects Protalix’s Enzyme Replacement Therapy for Rare Genetic Disease
2021-04-29
AnaptysBio’s Dermatology Drug Hits Roadblock in Phase 2 Trial
2021-03-09
BridgeBio Bags First FDA Nod with Rare Disease Drug Acquired from Alexion
2021-03-03
Researchers Test Gene Augmentation Therapy in Patient-Derived Retinal Organoids to Find Eye Disease Cure
2021-02-23
Horizon Buys AstraZeneca Spinout for $3 Billion to Broaden Rare Disease Pipeline
2021-02-02
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LATEST
Vaccine Protecting Against 20 Different Pneumococcal Infections Proves Effective in Trial
2022-08-12
FDA Approves Roche’s Established Antiviral For Children Aged Five Or Older
2022-08-12
Novartis Reported Two Deaths Following Zolgensma Gene Therapy
2022-08-12
Extreme Climate Change Is Aggravating Infectious Diseases!
2022-08-11
In Response to Expanding Monkeypox Outbreak, FDA Approves Emergency Use of JYNNEOS Vaccine
2022-08-11
Potential $1 billion Acquisition of TeneoTwo by AstraZeneca comes to Fruition
2022-08-11
Gene Repair Therapy: First Patient Dosed With New Treatment for Sickle Cell Disease
2022-08-11
EVENT
2022-09-09
ESMO 2022 Exhibition
Online and on-side
2022-09-20
Cell & Gene Therapy USA 2022
Virtual
2022-09-21
CSCO Educational Book 2022
Xiamen
2022-09-21
RESI Boston
Boston
2022-09-26
Human Gene Therapy Conference
Australia
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