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Kyowa Kirin Responds to NICE’s Publication of Appraisal Document for CRYSVITA

Kyowa Kirin Responds to NICE’s Publication of Appraisal Document for CRYSVITA
BUSINESSWIRE
29 Nov 2023
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FDA Approves First Therapy for Rare Type of Non-Cancerous Tumors

FDA Approves First Therapy for Rare Type of Non-Cancerous Tumors
FDA
28 Nov 2023
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Takeda aligns with leading rare disease group

Takeda aligns with leading rare disease group
Beth Snyder Bulik ENDPTS
28 Nov 2023
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FDA open to using single-arm studies for accelerated approval of gene therapies

FDA open to using single-arm studies for accelerated approval of gene therapies
Lei Lei Wu ENDPTS
27 Nov 2023
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Sernova Receives Rare Pediatric Disease Designations for Hemophilia A Program

Sernova Receives Rare Pediatric Disease Designations for Hemophilia A Program
GLOBENEWSWIRE
27 Nov 2023
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Mesoblast Files for Pediatric Rare Disease Designations for Rexlemestrocel-L

Mesoblast Files for Pediatric Rare Disease Designations for Rexlemestrocel-L
GLOBENEWSWIRE
26 Nov 2023
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Medicure Receives Approval to Enroll Patients for Treatment of Rare Disease

Medicure Receives Approval to Enroll Patients for Treatment of Rare Disease
ACCESSWIRE
23 Nov 2023
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Patient foundation accuses rare disease company Taysha of violating agreement

Patient foundation accuses rare disease company Taysha of violating agreement
ENDPTS
16 Nov 2023
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One Biosciences Partners With AP-HP to Launch a Single-cell Research Project

One Biosciences Partners With AP-HP to Launch a Single-cell Research Project
GLOBENEWSWIRE
10 Nov 2023
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Government plans to offer support for treatment of rare diseases

Government plans to offer support for treatment of rare diseases
ECONOMIC TIMES
07 Nov 2023
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