2026-04-17 • seekingalpha.com
Amicus Acquisition Boosts BioMarin Pharmaceutical’s Rare Disease Portfolio Making It a Buy
2026-04-17 • seekingalpha.com
Amicus Acquisition Boosts BioMarin Pharmaceutical’s Rare Disease Portfolio Making It a Buy2026-04-16 • EIN Presswire
Michele Levoir Sloan Recognized as Key Figure by Influential Women for Her Work Leading a Nonprofit in Rare Disease Advocacy2026-04-16 • prnewswire.co.uk
myTomorrows collaborates with Rare Disease Research to enhance access to rare disease clinical trials2026-04-16 • theparliamentmagazine.eu
European biotech innovation needs to result in quicker treatment options for individuals with uncommon illnesses2026-04-16 • manilatimes.net
Orvida Pharma formerly Kamari Pharma reports positive Phase 1a safety results for KM-023 a novel oral TRPV3 inhibitor and begins Phase 1b trials for Olmsted Syndrome patients2026-04-16 • raps.org
Høeg highlights FDA initiatives to accelerate access to treatments for uncommon illnesses2026-04-15 • PR Newswire
Lunai Bioworks and Geneial have executed a Letter of Intent to create patient groups for uncommon illnesses to support collaborations with pharmaceutical companies.2026-04-15 • thepharmaletter.com
Filspari secures historic US authorization for Travere2026-04-15 • businesswire.com
Savara Will Share Updated Information at the American Thoracic Society’s 2026 Conference2026-04-15 • pharmiweb.com
U.S. FDA Awards RMAT Status to Grace Science’s GS-100 Gene Therapy for NGLY1 Deficiency2026-04-15 • pharmiweb.com
Lantern Pharma introduces Zeta.ai a groundbreaking A.I. co-scientist designed for rare cancer drug discovery and development marking the world’s first multi-agentic platform; subscriptions are now available with2026-04-15 • showme.missouri.edu
University of Missouri’s substitute player success aids a novel medication treating a debilitating illness lacking a treatment option2026-04-14 • PR Newswire
Illumina partners with the Center for Data-Driven Discovery in Biomedicine to provide genomic data and software for battling pediatric cancer and rare diseases2026-04-09 • grafa.com
FDA approves Mesoblast to proceed with key DMD trial2026-04-09 • manilatimes.net
Climb Bio will present an R&D webcast showcasing Budoprutug and the CD19 potential on May 5, 20262026-04-09 • mychesco.com
Palvella Announces 2025 Performance and Progress on QTORIN Drug Development2026-04-09 • businesswire.com
KalVista Pharmaceuticals will showcase at the 25th Annual Needham Virtual Healthcare Conference2026-04-09 • manilatimes.net
Mesoblast Gains FDA IND Approval to Directly Enter Registration Trial for Ryoncil® for Duchenne Muscular Dystrophy2026-04-09 • in.investing.com
FDA approves REYOBIQ as an orphan drug for Plus Therapeutics2026-04-09 • nationaltoday.com
AIRNA Initiates Phase 1 Trial for Novel RNA Editing Treatment2026-04-09 • mychesco.com
Ocugen Finishes Dosing Phase in Stargardt Disease Gene Therapy Study2026-04-09 • investor.insmed.com
Insmed Reports Clinical Findings from Phase 2b CEDAR Trial2026-04-09 • nationaltoday.com
Oregon Health Science University Broadens Study to Combat Cancer in Uncommon Genetic Disease2026-04-09 • raps.org
The FDA’s budget plan includes a novel clinical trial route and continued approval for the pediatric disease voucher program.2026-04-09 • globenewswire.com
Plus Therapeutics Receives FDA Orphan Drug Status for REYOBIQ™ in Childhood Brain Tumors2026-04-08 • clinicaltrialsarena.com
Amgen announces favorable Phase III results for injectable Tepezza study2026-04-07 • quiverquant.com
Opus Genetics Receives $155 Million in Funding from Oberland Capital for Gene Therapy Development2026-04-07 • pharma.economictimes.indiatimes.com
The uncommon illness support group is requesting the Trump administration to reinstate FDA definition.2026-04-07 • yahoo.com
Glycomine completes Phase IIb POLAR trial participant recruitment2026-04-07 • biospace.com
The FDA’s changes regarding rare diseases reveal uncertainty concerning outside oversight.39 minutes ago • prnewswire.co.uk
Toby Neugebauer Incredibly Bullish on Project Matador and Calls for Sale of Company to Maximize Shareholder Value46 minutes ago • investorwire.com
Massimo Group (NASDAQ: MAMO) Appoints Quenton Petersen as CEO, Transitions David Shan to Executive Chairman46 minutes ago • biomedwire.com
Oncotelic Therapeutics, Inc. (OTCQB: OTLC) is highlighted in a BioMedWire editorial discussing the biotech industry’s move to base asset valuations on scientific evidence.46 minutes ago • techmediawire.com
Wearable Devices Ltd announces planned augmented reality collaboration with Meta-Bounds prior to the AWE 2026 presentation46 minutes ago • biomedwire.com
Oragenics Inc. shows early dosing advancement during Phase IIa trial for nasal concussion treatment.46 minutes ago • networknewswire.com
Brean Capital Initiates Coverage of OptimumBank Holdings, Inc. with a Buy Rating and $8 Price Target47 minutes ago • defensewirenews.com
SPARC AI Inc. partners with a second Ukrainian drone manufacturer for its Overwatch platform56 minutes ago • miningnewswire.com
Nevada Organic Phosphate Inc. initiating drilling preparations at the Murdock Mountain Project56 minutes ago • miningnewswire.com
ESGold Corp announced drilling at the Santa Fe Project targeting historic heap leach pads.3 hours ago • prnewswire.co.uk
JA Solar Summit Highlights Shift Toward Solar-Storage Integration as Global Demand Holds Firm3 hours ago • prnewswire.co.uk
Thunes Launches Real-Time Payments into New Zealand11 hours ago • prnewswire.co.uk
The Zero Waste Foundation has placed Türkiye’s COP31 strategy at UN Meetings in New York.46 minutes ago • techmediawire.com
Wearable Devices Ltd announces planned augmented reality collaboration with Meta-Bounds prior to the AWE 2026 presentation39 minutes ago • PR Newswire
New Alzheimer's Association Report: Americans Care Deeply About Brain Health but Most Don't Know How to Maintain It39 minutes ago • prnewswire.co.uk
Toby Neugebauer Incredibly Bullish on Project Matador and Calls for Sale of Company to Maximize Shareholder Value15 hours ago • EIN Presswire
Landmark Capital Advisors Reports Changing Trends in Indian Real Estate Investment18 hours ago • PR Newswire
Shriram Finance Earns Recognition as One of India’s Top Workplaces™ for Health & Wellness in 2026, Marking a Third Year of Achievement20 hours ago • EIN Presswire
Yudiz Version 2.0 Represents a Major Change for Digital Product Development in India18 hours ago • EIN Presswire
Top cyclists preparing for a major battle at the London 3 Day event in 2026 – the sole UK competition is confirmed18 hours ago • EIN Presswire
Angelfish Marketing Broadens AEO and AI Search Offerings for Business-to-Business Companies19 hours ago • prnewswire.co.uk
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