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News about "rare disease"
US FDA Accepts BioMarin's sNDA for VOXZOGO in Achondroplasia
The US Food and Drug Administration (FDA) has accepted BioMarin's sNDA for VOXZOGO in achondroplasia, supported by long-term safety and efficacy data, including adult height, body proportionality, and arm span outcomes.
Rare Disease | 14/07/2026 | By News Bureau
Ionis Completes Enrollment in Phase 3 REVEAL Study for Angelman Syndrome
Ionis has completed enrollment in the pivotal Phase 3 REVEAL study of obudanersen for Angelman syndrome, with topline clinical trial results expected in the second half of 2027.
Rare Disease | 06/07/2026 | By Akanki | 238
Chugai Pharma's Avastin Secures Approval in Japan for Additional Indication
Chugai Pharma’s Avastin has received approval in Japan for an additional indication, becoming the world’s first therapeutic option for neurofibromatosis type 2. The approval is supported by positive phase 2 clinical study results.
Rare Disease | 22/06/2026 | By News Bureau
Regel Therapeutics Names Stephen J. Farr as New CEO
Regel Therapeutics’ Board of Directors has named Stephen J. Farr, PhD, as the new Chief Executive Officer (CEO).
Rare Disease | 21/06/2024 | By Aishwarya | 430
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