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Amylyx Pharmaceuticals to Present Updates on its AMX0114 and RELYVRIO® Programs in ALS at the 2024 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference
2/15/2024
Amylyx Pharmaceuticals, Inc. announced that two abstracts detailing next steps in the development of the Company’s investigational antisense oligonucleotide, AMX0114, for the potential treatment of amyotrophic lateral sclerosis and findings from a pharmacokinetic and pharmacodynamic study of AMX0035 in people with ALS will be presented at the Muscular Dystrophy Association Clinical and Scientific Conference on March 3-6, 2024 in Orlando, Florida.
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AusperBio Announces Major Progress in AHB-137 Clinical Development
2/14/2024
AusperBio Therapeutics, Inc. and Ausper Biopharma Co., Ltd. announced significant updates on the clinical development of its leading molecule, AHB-137, an unconjugated antisense oligonucleotide targeting all HBV RNA.
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Dyne Therapeutics Announces Upcoming Presentations on Initial Clinical Data From its ACHIEVE Trial in DM1 Patients and DELIVER Trial in DMD Patients at the 2024 Muscular Dystrophy Association Clinical & Scientific Conference
2/14/2024
Dyne Therapeutics, Inc. announced two oral presentations at the Muscular Dystrophy Association Clinical & Scientific Conference being held March 3-6, 2024, in Orlando, Florida and virtually.
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Quest Diagnostics Debuts MelaNodal Predict™ Test, Personalizing Melanoma Risk Prediction to Help Patients Forgo Invasive Surgery
2/13/2024
Quest Diagnostics announced the launch of MelaNodal Predict™, a highly advanced predictive gene expression test to help personalize treatment decisions for patients with melanoma, the deadliest form of skin cancer and one of the most common cancers in the United States.1 Dermatologists and other providers nationwide may begin ordering the test today from Quest Diagnostics, as well as through Dermpath Diagnostics, Quest's subspecialty pathology business.
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RTW Charitable Foundation Announces Six Research Grant Recipients
2/12/2024
RTW Charitable Foundation announced that it has awarded $900,000 in research grants to support six research studies.
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Rapafusyn's Rick Ewing Acknowledged with Dual Honors for Exceptional Achievements
2/5/2024
Baltimore, MD – February 1, 2024 – Rapafusyn proudly announces the distinguished recognition of Dr. Rick Ewing, Vice President of Medicinal Chemistry, for his outstanding contributions to the realms of chemistry and biotechnology innovation.
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Praxis Precision Medicines, Inc. Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4) - February 02, 2024
2/2/2024
Praxis Precision Medicines, Inc. announced that on February 1, 2024, the Compensation Committee of Praxis’ Board of Directors granted non-qualified stock option awards to purchase an aggregate of 1,600 shares of its common stock and restricted stock unit awards covering an aggregate of 1,450 shares of its common stock to two new non-executive employees under the Praxis Precision Medicines, Inc. 2024 Inducement Plan.
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iXCells Biotechnologies Announces Grand Opening and 2024 Rare Disease Month Workshop
2/1/2024
iXCells Biotechnologies USA, Inc. announced the grand opening celebration of its new San Diego headquarters and Rare Disease Month Workshop.
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Biogen to Realign Resources for Alzheimer's Disease Franchise
1/31/2024
Biogen Inc. (Nasdaq: BIIB) today announced plans to reprioritize its resources in Alzheimer’s disease (AD), a strategic therapeutic area expected to drive near and long-term growth.
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AcuraStem Secures $7 Million in Funding Support from NIH and DOD to Accelerate Therapies for ALS and FTD
1/30/2024
AcuraStem, a patient-based biotechnology company advancing treatments for amyotrophic lateral sclerosis, frontotemporal dementia, and other neurodegenerative diseases, announced that it has successfully raised nearly $7 million in grant funding for ALS / FTD research from the National Institutes of Health and the Department of Defense.
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Vanda Pharmaceuticals Receives FDA Approval to Proceed with Investigational New Drug VCA-894A, a Novel Antisense Oligonucleotide Candidate for the Treatment of Charcot-Marie-Tooth Disease, Type 2S
1/23/2024
Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced that the U.S. Food and Drug Administration (FDA) has approved the Investigational New Drug (IND) application to evaluate VCA-894A for the treatment of a patient with Charcot-Marie-Tooth disease.
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Stoke Therapeutics Announces Inducement Grants Under Nasdaq Listing Rule 5635(C)(4)
1/17/2024
Stoke Therapeutics, Inc. announced that, effective on January 16, 2024, the company granted stock options to purchase an aggregate of 13,300 shares of common stock to 2 new employees, as a material inducement to their employment in accordance with Nasdaq Listing Rule 5635.
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Stoke Therapeutics Highlights Strategic Priorities and Anticipated Milestones for 2024
1/8/2024
Stoke Therapeutics, Inc. announced strategic priorities and anticipated milestones for 2024.
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Praxis Precision Medicines Provides Update on Advancing Clinical Stage Portfolio
1/8/2024
Praxis Precision Medicines, Inc. provided an update on its portfolio and planned key milestones in 2024.
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Denali Therapeutics Announces Key Anticipated 2024 Milestones and Priorities to Further Advance Its Therapeutics Portfolio for Neurodegeneration and Lysosomal Storage Diseases
1/8/2024
Denali Therapeutics Inc. (Nasdaq: DNLI) today announced program progress and expected milestones for 2024, which Chief Executive Officer, Ryan Watts, Ph.D., will highlight during a corporate presentation at the 42nd Annual J.P. Morgan Healthcare Conference on Tuesday, January 9th, at 10:30 a.m. Pacific Time.
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Praxis Precision Medicines Announces Licensing and Collaboration Agreement with Tenacia Biotechnology for Ulixacaltamide in Greater China
1/5/2024
Praxis Precision Medicines today announced an exclusive collaboration and license agreement with Tenacia Biotechnology (Shanghai) Company, Ltd., a China-based clinical-stage biopharmaceutical company focused on CNS drug development and commercialization.
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Vico Therapeutics Announces $60 million (€54 million) Series B Financing to Advance VO659 Clinical-Stage Program for Rare Neurological Diseases and Expand Pipeline
1/5/2024
Vico Therapeutics B.V. announced a $60 million Series B financing.
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Stoke Therapeutics to Present at the 42nd Annual J.P. Morgan Healthcare Conference
1/4/2024
Stoke Therapeutics, Inc. announced that management will present at the 42nd Annual J.P. Morgan Healthcare Conference on Wednesday, January 10, 2024, at 6:45 p.m. ET.
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Dyne Therapeutics Announces Positive Initial Clinical Data from ACHIEVE Trial in DM1 Patients and DELIVER Trial in DMD Patients Demonstrating Promise of the FORCE™ Platform in Developing Therapeutics for Rare Muscle Diseases
1/3/2024
Dyne Therapeutics, Inc. announced positive initial clinical data from its ACHIEVE trial of DYNE-101 in patients with myotonic dystrophy type 1 and its DELIVER trial of DYNE-251 in patients with Duchenne muscular dystrophy who are amenable to exon 51 skipping.
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Matinas BioPharma to Present at Biotech Showcase 2024
1/3/2024
Matinas BioPharma announces that Jerome D. Jabbour, Chief Executive Officer, will present a company overview at the Biotech Showcase 2024 on Tuesday, January 9, 2024 at 11:00 a.m. Pacific time.