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Genetic Disorder Equipment Supplied In Usa
17 equipment items found
Manufactured by:Access Bio based inSomerset, NEW JERSEY (USA)
Recommended by the WHO during the Malaria Policy Advisory Meeting. G6PD deficiency is an X-linked recessive genetic disorder, resulting in no or low G6PD activity. G6PD deficienct patients are vulnerable to the spontaneous destruction of red blood cells when exposed to high oxidative stress. High oxidative stress may result from consumption of primaquine ...
Manufactured by:Sana Biotechnology based inSeattle, WASHINGTON (USA)
Liver-related genetic disorders: Ornithine transcarbamylase deficiency ...
by:Cartesian Therapeutics based inGaithersburg, MASSACHUSETTS (USA)
ImmTOR has the potential to administer multiple low doses to achieve therapeutic benefit without risk of overdosing, the ability to treat patients typically excluded from these treatments and is a novel approach to treating systemic diseases resulting from genetic disorders. ...
by:Cartesian Therapeutics based inGaithersburg, MASSACHUSETTS (USA)
Selecta’s wholly owned gene therapy program for the treatment of ornithine transcarbamylase (OTC) deficiency is expected to enter the clinic in 2022. OTC deficiency is a genetic disorder urea cycle that causes ammonia to accumulate in the blood. The most severe form of the disorder presents within the first few days of life. Severe symptoms ...
Manufactured by:Rocket Pharmaceuticals, Inc. based inCranbury, NEW JERSEY (USA)
FA is a rare, genetic disorder affecting DNA repair. Approximately two-thirds of FA cases are caused by genetic defects in the FANCA gene, which results in the FA subtype known as FA Complementation Group A (FA-A). FA patients may develop bone marrow failure (very low blood counts), cancers of the blood or other ...
Manufactured by:GenSol Diagnostics based inClayton, GEORGIA (US) (USA)
Laryngeal paralysis (LP) is a genetic disorder that results in breathing difficulties which can be made worse by physical activity and may lead to suffocation in severe cases. The condition may require surgery to alleviate breathing difficulties. Symptoms of the disease can include a respiratory murmur, wheezing, reduced tolerance for exercise, voice impairment, ...
by:Neoclease based inBoston, MASSACHUSETTS (USA)
These enzymes are meticulously crafted using proprietary algorithms powered by state-of-the-art GPT neural networks, which are capable of redesigning nucleases for specific genetic targets. This approach allows Neoclease to tailor solutions uniquely to each client's needs. The focus on innovation extends to the creation of comprehensive nuclease libraries, ensuring a wide range ...
Manufactured by:Rocket Pharmaceuticals, Inc. based inCranbury, NEW JERSEY (USA)
LAD-I is a rare genetic disorder affecting the immune system and is caused by a defect in the ITGB2 gene, leading to a deficiency in CD18. Due to this deficiency, white blood cells (primarily neutrophils) are unable to leave the bloodstream to go to the site of infection. ...
Manufactured by:Rocket Pharmaceuticals, Inc. based inCranbury, NEW JERSEY (USA)
Danon Disease (DD) is a rare genetic disorder that is characterized by severe and primarily hypertrophic cardiomyopathy. Skeletal muscle weakness and mild cognitive impairment are also common. The causative mutation has been identified in the gene encoding for lysosome-associated membrane protein (LAMP2), and in particular the LAMP2B version of the gene which is ...
Manufactured by:HemoShear Therapeutics, Inc. based inCharlottesville, VIRGINIA (USA)
HST5040 is an investigational small molecule therapy being developed by HemoShear to reduce the levels of toxins associated with methylmalonic acidemia (MMA) and propionic acidemia (PA), rare genetic disorders caused by the de?ciency of certain enzymes required to metabolize amino acids. HST5040 is formulated for convenient daily administration at home as a ...
by:Aegle Therapeutics based inWoburn, MASSACHUSETTS (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. ...
Manufactured by:AVITA Medical based inValencia, CALIFORNIA (USA)
Epidermolysis Bullosa (EB) is a group of rare and incurable skin disorders caused by mutations in genes encoding structural proteins resulting in skin fragility and blistering, leading to chronic wounds and, in some sub-types, an increased risk of squamous cell carcinoma or death. Dystrophic EB is estimated to affect 3-8 million people[1], and there is currently no FDA-approved ...
Manufactured by:Rocket Pharmaceuticals, Inc. based inCranbury, NEW JERSEY (USA)
PKD is a rare, genetic blood disorder. PKD is caused by a defect in the PKLR gene which is responsible for energy production in red blood cells (RBCs). RBCs carry oxygen to the rest of the body. When the PKLR gene has a defect, RBCs are limited in their ability to produce energy and remain intact. ...
Manufactured by:Aalto Scientific, Ltd. based inEatonton, GEORGIA (US) (USA)
Homocysteine Control is a stable liquid product intended to simulate human patient samples for the purpose of monitoring the precision of laboratory testing procedures for ...
by:Intellia Therapeutics, Inc. based inCambridge, MASSACHUSETTS (USA)
The first patient was dosed with our investigational genome editing treatment for transthyretin (ATTR) amyloidosis in November 2020. Our modular approach enables us to optimize the power and versatility of the CRISPR/Cas9 technology and, importantly, allows us to rapidly develop therapeutics for numerous diseases that currently have limited treatment ...
Manufactured by:Ocugen, Inc. based inMalvern, PENNSYLVANIA (USA)
OCU400 (AAV-NR2E3) is a novel gene therapy product candidate with the potential to be broadly effective in restoring retinal integrity and function across a range of genetically diverse inherited retinal diseases (“IRDs”). It consists of a functional copy of a nuclear hormone receptor (“NHR”) gene, NR2E3, delivered to target cells in the retina using an adeno-associated ...
by:Neurona Therapeutics based inSouth San Francisco, CALIFORNIA (USA)
Neurona’s lead product candidate, NRTX-1001, comprises human MGE-type inhibitory GABAergic interneurons derived from pluripotent stem cells. NRTX-1001’s first indication is for the treatment of drug-resistant focal epilepsy, which is a major unmet need affecting approximately one-third of people living with epilepsy. Epileptic seizures are characterized by imbalanced neural activity ...
