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Rare Disease Articles & Analysis
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We look forward to continuing our collaborations and partnerships, contributing to advancements in drug development and improving patient outcomes." mPEG-pALD (20K) plays a vital role in various applications, including the development of therapeutic protein drugs, biosimilars, rare disease treatments, and advanced biomedical research. Additionally, it is ...
Protheragen, a dedicated preclinical research service provider specializing in rare diseases, announces its comprehensive diagnostic development services aimed at empowering researchers in the realm of rare disease research and drug development. These diagnostic services are designed to support the scientific community in ...
Protheragen is proud to release the launch of its comprehensive vaccine development solutions tailored to advance research and development in the field of infectious diseases. This strategic initiative aims to accelerate the creation of effective vaccines to combat emerging and re-emerging infectious threats. Infectious diseases are disorders caused by organisms — such as bacteria, ...
SOPHiA GENETICS (Nasdaq: SOPH), a cloud-native healthcare technology leader in data-driven medicine, today announced the next step in the partnership between the Company and Genomenon, a leading genomic intelligence company, to support better, data-driven outcomes in the rare disease and oncology fields. The two companies have expanded the integration of ...
Influenza A virus strains of all subtypes have been isolated from wild birds, but disease is rare. Some isolated influenza A viruses cause severe disease in poultry and, in rare cases, in humans. ...
We believe that by using the same cells that promote tissue repair, organ maintenance, and immune system function, we can develop safe and effective therapies for some of the most difficult diseases and conditions. In 2024, we are focusing our efforts on two of our most promising programs: Hypoplastic Left Heart Syndrome (HLHS); and Alzheimer's Disease. HLHS: ...
As several gene therapy approvals for rare disease treatment gain steam in 2023, a comprehensive set of gene therapy development solutions is released to pave richer avenues for rare disease research. This path-breaking move sees the integration of cutting-edge technology with state-of-art research facilities to amplify the ...
While IVS currently focuses on applications of the LITATM in cardiac safety and discovery projects, IVS has plans for developing a wide range of useful applications in treatment development for regenerative medicine, cancer, autoimmune disorders, and a wide range of rare diseases. Our unique combination of tissue engineering technology with assay automation and ...
These skills can boost your career whether you specialize in oncology, immunology, cardiology, neurology, diabetes, or any other rare disease and therapeutic area. Are you looking for high-performing and diverse millennials to join your team? ...
TMAs, such as aHUS, are rare diseases primarily affecting the kidney. They are hard to diagnose using traditional methods as they present as a number of nonspecific symptoms, such as severe hypertension, acute kidney injury/failure in the context of mechanical anemia and thrombocytopenia. ...
A five-year action plan developed to meet requirements under Section 4 of the Accelerating Access to Critical Therapies for ALS Act. ...
His career has focused on technology development and the application of genomics to cancer biology and human diseases, with research in autism, ADHD, vertigo, brain cancers, rare disease diagnostics and therapeutics. ...
TGen is part of City of Hope, a world-renowned independent research and treatment center for cancer, diabetes and other life-threatening diseases. This precision medicine affiliation enables both institutes to complement each other in research and patient care, with City of Hope providing a significant clinical setting to advance scientific discoveries made by TGen. TGen is ...
Oligomerix, Inc., a privately held company pioneering the development of small molecule therapeutics targeting tau for Alzheimer’s and rare neurodegenerative diseases, announced that the Company presented data from a therapeutic study in an animal model of its lead product candidate OLX-07010 at the 14th Annual Clinical Trials on Alzheimer's ...
Among these areas are oncology, immunology, rare diseases, vaccines, potential cures in other core areas of Sanofi’s business footprint, and digital health solutions. 1 Wei Y, Ren X, Galbo PM Jr, et al. ...
ByBayer AG
Commercial and Pipeline Update Product portfolio Acute Care Hospital: RYANODEX®, vasopressin, Barhemsys®, Byfavo® Oncology: BENDEKA, BELRAPZO, PEMFEXY11, TREAKISYM Japan12 75-person commercial team covers all products excluding BENDEKA and TREAKISYM Company projects growth in earnings while still supporting R&D Cash flow from legacy products expected to continue to ...
LB-P8 is, to the best of company's knowledge, the first drug to be granted orphan drug designation in the field of microbiome-based treatment for metabolic diseases. The US FDA's ODD program supports the development of treatments for rare diseases with high unmet medical needs and provides various incentives. ...
(“BRIM,” TPEx 6885) is pleased to announce that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for BRM424 in the treatment of neurotrophic keratitis (NK), a rare degenerative eye disease which causes very severe cornea damage and can lead to loss of sight. ...
(NASDAQ:LUMO), a clinical-stage biopharmaceutical company focused on therapeutics for rare diseases, announced that the Company will present at the H.C. Wainwright Virtual BIOCONNECT Conference being held January 10-13, 2022. ...
Phase 1 studies show improvement in cognitive factors, consistent with therapeutic potential of CY6463 to improve key features in neurological diseases CY6463 favorable tolerability and safety profile was confirmed Ariana Pharma, a leading Artificial Intelligence (AI) drug development company, in collaboration with Cyclerion Therapeutics, presented today safety and ...