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...
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 ...
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.)...
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...
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...