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CENTOGENE and Agios Expand Partnership for Clinical Development of PYRUKYND® (mitapivat) to Treat Children With Rare Blood Disease
June 13, 2022 06:30 ET | Centogene NV
Expanded collaboration to include Phase 3 ACTIVATE-kids and ACTIVATE-kidsT studies CENTOGENE’s genetic testing and Biodatabank identify causative mutations, including the UGT1A1 and PKLR genes, and...
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Ultragenyx Announces Positive Longer-term Durability Data from Two Phase 1/2 Gene Therapy Studies at American Society of Gene & Cell Therapy (ASGCT) 2022 Annual Meeting
May 19, 2022 08:00 ET | Ultragenyx Pharmaceutical Inc.
Durable and clinically meaningful responses sustained for more than three years post-treatment in study of DTX401 for GSDIa and four or more years in study of DTX301 for OTC NOVATO, Calif., May ...
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Ultragenyx Acquires Global Rights to AAV Gene Therapy ABO-102 for Sanfilippo Syndrome Type A (MPS IIIA) from Abeona Therapeutics
May 17, 2022 08:00 ET | Ultragenyx Pharmaceutical Inc.
ABO-102 / UX111 is being evaluated in ongoing pivotal Transpher A trial in patients with MPS IIIA Interim data featured in encore oral presentation at American Society of Gene & Cell Therapy...
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Decibel Therapeutics Reports First Quarter 2022 Financial Results and Corporate Update
May 12, 2022 07:30 ET | Decibel Therapeutics, Inc.
- Multiple milestones anticipated for 2022 including submission of IND and/or CTA for DB-OTO, top-line results from interim analysis of Phase 1b trial of DB-020 for cisplatin-induced hearing loss and...
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Catalyst Pharmaceuticals Reports Record First Quarter 2022 Financial Results And Provides Corporate Update
May 10, 2022 16:11 ET | Catalyst Pharmaceuticals, Inc.
Achieved Record Revenues of more than $43 Million, a 42.7% Increase YoY Three New Patents Issued; Now Five Orange-Book Listed Patents for FIRDAPSE® Significant Cash Position of $198 Million and...
Streaming Channel Teams Up With Award-Winning Filmmaker to Debut Series About Life With Long Covid Plus Rare Disease
May 10, 2022 09:00 ET | The Disorder Channel
FALMOUTH, Maine, May 10, 2022 (GLOBE NEWSWIRE) -- The Disorder Channel, a free streaming channel for rare disease stories on TV, is uniting with nonprofit The Solve ME/CFS Initiative (Solve M.E.)...
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CENTOGENE and Takeda Extend Market Access and Expansion Partnership to Advance Patient Care Globally
April 28, 2022 06:30 ET | Centogene NV
Accelerating the path from diagnosis to available treatments for rare metabolic and rare neurodegenerative diseases CENTOGENE and Takeda extend contract to continue providing access to genetic...
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ReAlta Life Sciences, Inc. Partners With Hope for HIE to Improve Neonatal Brain Injury Outcomes and Quality of Life
April 27, 2022 09:30 ET | Hope for HIE
WEST BLOOMFIELD TOWNSHIP, Mich., April 27, 2022 (GLOBE NEWSWIRE) -- ReAlta Life Sciences, Inc. ("ReAlta") a clinical stage, rare disease company addressing life-threatening diseases through...
Saniona Announces Refocused Strategy on Ion Channel R&D and Restructuring of Board of Directors and Executive Management Team
April 25, 2022 10:25 ET | Saniona AB
PRESS RELEASE April 25, 2022 Jørgen Drejer, co-founder and board member, appointed interim Chairman of the Board of Directors; Thomas Feldthus, co-founder, appointed as CEO; Anita Milland...
Saniona tillkännager nytt strategiskt fokus på jonkanalsrelaterad FoU samt omstrukturering av styrelse och ledningsgrupp
April 25, 2022 10:25 ET | Saniona AB
PRESSMEDDELANDE 25 april 2022 Jørgen Drejer, medgrundare och styrelseledamot, kommer att föreslås till styrelseordförande; Thomas Feldthus, medgrundare, utses till VD; Anita Milland utses till...