NIH scientists pursue new therapies to improve rare disease drug development

(NIH/National Center for Advancing Translational Sciences (NCATS)) Four new preclinical drug development projects at the National Institutes of Health will target a form of blindness and diseases characterized by cardiac problems. The projects were selected for their potential to treat specific rare diseases and to help scientists uncover new information that can be shared with other researchers.
Source: EurekAlert! - Medicine and Health - Category: Global & Universal Source Type: news

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Gyroscope Therapeutics, a retinal gene therapy company, has developed the Orbit subretinal delivery system (SDS). The technology is designed to deliver therapies to the retina without the need for invasive procedures, such as vitrectomies, which invo...
Source: Medgadget - Category: Medical Devices Authors: Tags: Exclusive Genetics Medicine Source Type: blogs
Basel, 10 September 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) will present new ENSPRYNG ® (satralizumab) data on reducing relapse severity in the treatment of neuromyelitis optica spectrum disorder (NMOSD), a rare disease of the central nervous system. These data are being presented at MSVirtual2020, the 8th joint ACTRIMS-ECTRIMS meeting, in addition to longer-term efficacy data supporting the continued effect of ENSPRYNG on reducing the risk of NMOSD relapse, as well as its favourable benefit:risk profile.“The data for ENSPRYNG at MSVirtual2020 are promising and suggest it significantly reduces relapse severity ...
Source: Roche Media News - Category: Pharmaceuticals Source Type: news
Early blindness secondary to incurable retinal detachment is one of the main complications of incontinentia pigmenti (IP). The efficiency of ophthalmological management for preventing such evolution has not been ...
Source: Orphanet Journal of Rare Diseases - Category: Internal Medicine Authors: Tags: Research Source Type: research
Conclusions: This research provides an overview of symptoms experienced by patients with USH1 and highlights the dramatic impact these have on patients' lives, allowing the identification of concepts of importance when evaluating therapeutic treatments in development for RP. PMID: 32367747 [PubMed - as supplied by publisher]
Source: Ophthalmic Genetics - Category: Opthalmology Tags: Ophthalmic Genet Source Type: research
The adult patient has a rare disease that causes blindness.
Source: bizjournals.com Health Care:Pharmaceuticals headlines - Category: Pharmaceuticals Authors: Source Type: news
The field of medical AI is buzzing. More and more companies set the purpose to disrupt healthcare with the help of artificial intelligence. Given how fast these companies come and go, it can prove to be hard to stay up-to-date with the most promising ones. Here, I collected the biggest names currently on the market ranging from start-ups to tech giants to keep an eye on in the future. To further help you keep up with what A.I. brings to medicine, The Medical Futurist team made an easy-to-digest e-book about just that. I highly encourage you to read it and would love to hear about your thoughts! Artificial Intelligence has ...
Source: The Medical Futurist - Category: Information Technology Authors: Tags: Artificial Intelligence Healthcare Design AI digital health genetics Innovation Personalized medicine pharma GC1 big data drug development healthcare companies medical imaging Source Type: blogs
Publication date: Available online 7 November 2019Source: Saudi Journal of OphthalmologyAuthor(s): Abdulrahman H. Badawi, Ahmed A. Al-Muhaylib, Adi Mohammed Al Owaifeer, Rakan S. Al-Essa, Sami A. Al-ShahwanAbstractPrimary congenital glaucoma (PCG) is a rare disease affecting children early in life. PCG was considered untreatable with inevitable blindness. However, recent advances in biochemical and genetic studies, the introduction of new diagnostic tools, intraocular pressure (IOP) lowering medications and improvement of surgical techniques have led to a better understanding of this devastating disease and preserving the ...
Source: Saudi Journal of Ophthalmology - Category: Opthalmology Source Type: research
AbstractAutoimmune uveitis is a sight-threatening, rare disease, potentially leading to blindness. Uveitis is a synonym for intraocular inflammation, presenting as various clinical phenotypes with different underlying immune responses in patients, whereas different animal models usually represent one certain clinical and immunological type of uveitis due to genetic uniformity and the method of disease induction. T cells recognizing intraocular antigens initiate the disease, recruiting inflammatory cells (granulocytes, monocytes/macrophages) to the eyes, which cause the damage of the tissue. The treatment of uveitis so far ...
Source: Seminars in Immunopathology - Category: Pathology Source Type: research
In this study, researchers studied 438,952 participants in the UK Biobank, who had a total of 24,980 major coronary events - defined as the first occurrence of non-fatal heart attack, ischaemic stroke, or death due to coronary heart disease. They used an approach called Mendelian randomisation, which uses naturally occurring genetic differences to randomly divide the participants into groups, mimicking the effects of running a clinical trial. People with genes associated with lower blood pressure, lower LDL cholesterol, and a combination of both were put into different groups, and compared against those without thes...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
Multivitamins, drugs, gene therapies, human skin, heart, eyeballs, kidneys, entire dead bodies – everything comes with a price tag. Putting aside the moral questions of why and how come that the capitalist market priced even our body parts and health, we asked the question of how much is life worth: what is the maximum that you would/should pay for a life-saving drug? How high is too high a cost if a drug can save 200-300 babies a year from debilitating illness or death? And ultimately, does the pricing of new technologies, especially gene therapies, enable to fulfill their promise? There’s a price for ever...
Source: The Medical Futurist - Category: Information Technology Authors: Tags: Bioethics Biotechnology Future of Pharma Genomics cost daraprim drug drug price Gene gene therapy genetics insulin life medication pricing policy rare disease rare disorder Source Type: blogs
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