Rare Disease Equipment
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by Healx Ltd.based in UNITED KINGDOM
The world’s most efficient AI platform for rare disease drug discovery, supporting our teams and partners in our mission to advance new treatments towards the ...
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Manufactured by InvivoSciences, Inc. (IVS)based in USA
InvivoSciences (IVS) combines its human micro tissue technology and phenotypic assay platform with the systems biology approach to achieve rare disease modeling for its drug discovery. Our strip tissues are perfect for modeling muscle and connective tissue in micro-well plates. Since many rare diseases affect heart muscle and ...
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based in USA
Epidermolysis bullosa (“EB”) is a group of rare genetic disorders that manifests as blistering or erosion of the skin in response to little or no apparent trauma. There are many genetic and symptomatic variations of EB. EB is always painful, often pervasive and debilitating, and is in some cases lethal before the age of 30. EB ...
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based in USA
There exist numerous rare diseases that tau dysfunction is associated with known as tauopathies. While Alzheimer’s is the largest tauopathy, other tauopathies are rare diseases that present accelerated regulatory pathways and significant near-term commercial opportunities. These rare ...
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based in USA
People with rare diseases often wait years to receive a proper diagnosis and many never receive one at all. RARE ANSWERS™ is a system of innovative and sustainable tools designed to help shorten the diagnostic journey and time to initiating treatment of children with a rare disease. Developed in ...
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Manufactured by Bausch Health Companies Inc.based in CANADA
Our diversified portfolio of well-established specialty pharmaceuticals targets several therapeutic areas, including epilepsy, migraines, depression, chronic pain, and rare diseases such as Huntington's disease. ...
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based in POLAND
Myasthenia gravis (MG) is an autoimmune condition classified as a rare disease. Accordingly, myasthenic patients lack effective treatment options, as this rare disease is not a primary interest of the pharmaceutical industry. However, considerable social and institutional support exists for the development of new therapies to ...
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by Healx Ltd.based in UNITED KINGDOM
Our Rare Treatment Accelerator helps academic, patient, and industry groups advance their rare disease drug redevelopment projects. By combining our team, expertise and financial resources with your research and insights, we can get treatments to patients ...
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based in POLAND
Neuromyelitis optica (NMO) is an neurological autoimmune condition classified as a rare disease. Because of this classification, NMO lacks effective treatment options, since it is not of primary interest to the pharmaceutical industry. However, considerable social and institutional support exists for developing new therapies for such ...
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Manufactured by Lipella Pharmaceuticals Inc.based in USA
created and developed within the published guidance of the National Institute of Health Rare Diseases Registry Program ...
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Manufactured by HTG Molecular Diagnostics, Inc.based in USA
The initial focus of HTG Therapeutics will be to identify development candidates targeting RNA or RNA modifying proteins, which could be relevant in areas such as oncology, immunology, diabetes, and rare disease. HTG’s EdgeSeq and Epi-EdgeSeq technology is a foundational element in our drug discovery programs. It enables a more ...
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Manufactured by Eyevensysbased in FRANCE
EYS606 is a novel non-viral gene therapy approach that was developed for the treatment of chronic non-infectious uveitis (CNIU). CNIU, which meets the criteria for a rare disease (affecting 250,000 patients in North America and Europe), is a sight-threatening immune-mediated inflammatory disease that causes inflammation in the eye that can ...
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based in USA
Selecta’s first ImmTORTM + gene therapy candidate MMA-101 for methylmalonic acidemia (MMA) is expected to enter clinical trials the end of 2021. MMA is a rare metabolic disease that may lead to metabolic acidosis and hyperammonemia and is associated with long-term complications including feeding problems, developmental delay, ...
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Manufactured by Twist Biosciencebased in USA
Twist Exome 2.0 is designed to detect rare and inherited diseases, as well as germline cancers. This panel’s high uniformity and low off-target rate deliver best-in-class sequencing efficiency, enabling quality data to be collected with less sequencing. With superior coverage of major genetic databases (RefSeq, CCDS, GenCode, Clinvar, ...
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Manufactured by Catalent, Incbased in USA
Gene therapy holds the promise of treating the unmet needs of patients who suffer from a rare genetic disease. An estimated 4,000 medical conditions are a result of gene disorders with no previous targeted treatments. Gene therapy offers new options away from the conventional symptomatic approach to disease treatment as well as provides ...
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Manufactured by BRIM Biotechnology, Inc.based in TAIWAN
Neurotrophic Keratitis is a rare, degenerative disease characterised by corneal sensitivity reduction, spontaneous epithelium breakdown, and impairment of corneal healing. Currently, there is only one drug, Oxervate (Cenegermin), approved by the US FDA and EMA in 2018. Eye health is crucial to quality of life and BRIM is advancing new ...
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Manufactured by BOSANA Medizintechnik GmbHbased in GERMANY
tipstim® is a completely new product to improve sensorimotor abilities of the hand in rehabilitation after brain damage, such as stroke, craniocerebral trauma but even other rare neurological diseases as e.g. Complex Regional Pain Syndrome (CRPS Type 1). tipstim® was developed in collaboration with the the ...
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Manufactured by Kangstem Biotech Co., Ltd.based in SOUTH KOREA
Kangstem Biotech is developing stem cell therapeutic products for patients suffering from rare and incurable diseases using umbilical cord blood-derived stem ...
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Manufactured by BioArctic ABbased in SWEDEN
Research to develop new antibodies for treating neurodegenerative disorders is under way at BioArctic. the ND3014 project aims to develop selective antibody treatments targeting TDP-43, a protein that is believed to play a crucial role in the development of the rare neurodegenerative disease ALS (amyotrophic lateral ...
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Manufactured by HemoShear Therapeutics, Inc.based in USA
at home as a liquid taken either orally or through a gastrostomy tube. The FDA has granted HST5040 Orphan Drug, Fast Track and Rare Pediatric Disease designations for the treatment of MMA and ...
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