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Rare Disease
M&A Rare Disease
Horizon Buys AstraZeneca Spinout for $3 Billion to Broaden Rare Disease Pipeline
2021-02-02
Trials & Approvals Rare Disease
Orladeyo’s FDA approval Heralds New Funding for BioCryst
2020-12-13
M&A Rare Disease
AstraZeneca to Acquire Alexion for US$ 39 Billion to Grow Immunology Pipeline
2020-12-13
Trials & Approvals Pyruvate Kinase Deficiency
Agios Pharmaceuticals’ Lead Candidate Mitapivat Achieves Primary Endpoint in ACTIVATE Trial
2020-12-03
Trials & Approvals Liver Enzyme
Alnylam Receives FDA Nod for its Third Rare Disease Drug
2020-11-25
R&D Rare Disease
Retrophin Acquires Orphan Technologies to Develop Potential, Effective Cure for Rare Disease
2020-10-25
Trials & Approvals Rare Disease
Potential Treatment for Osteogenesis Imperfecta Receives FDA’s “Rare Pediatric Disease Designation”
2020-09-27
Trials & Approvals
Genentech Enters Spinal Muscular Atrophy Market with Risdiplam’s FDA Approval
2020-08-11
R&D
Sarepta and Codiak Biosciences Join Hands to Tackle Rare Diseases Using Exosome-Based Therapeutics
2020-06-26
R&D
Biogen’s SMA Drug, Spinraza Continues to Show Benefits in Pre-Symptomatic Patients
2020-06-14
Rare Disease
Daiichi Sankyo Invests in Ultragenyx’s Technology to Bolster Gene Therapy Pipeline
2020-04-02
R&D Rare Disease
Mochida Pharma, Gene Techno Science Collaborate to Develop Regenerative Medicine for Rare Disease
2020-03-28
Interviews Rare Disease
‘Rare diseases are not that rare’ Interview with Dr. Durhane Wong-Rieger
2019-11-08
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EVENT
2024-04-27
2024 Biomedical Final Pitch Competition
Room DA1620, Dana Building, Dana-Farber Cancer Institute, 99 Jimmy Fund Way, Boston, MA 02115
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