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Rare Disease Day 2017 With A Commitment To Provide New Insights Into Rare Diseases Equipment Supplied In Usa
24 equipment items found
Manufactured by:Twist Bioscience based inSouth San Francisco, CALIFORNIA (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, ACMG73 and more) and the addition of clinically relevant ...
by:Palvella Therapeutics. based inWayne, PENNSYLVANIA (USA)
QTORIN™ rapamycin 3.9% is a novel targeted topical therapy being studied for Microcystic Lymphatic Malformations (Micro LM). Palvella is enrolling a phase 2 clinical study for people with Micro ...
Manufactured by:Tonix Pharmaceuticals Holding Corp. based inChatham, NEW JERSEY (USA)
TNX-2900, Tonix’s proprietary potentiated intranasal oxytocin is in the pre-Investigational New Drug (IND) stage as a candidate for the treatment of Prader-Willi syndrome (PWS) and non-organic failure to thrive disease. TNX-2900 has been granted Orphan Drug Designation (ODD) in the US by the FDA for the treatment of ...
Manufactured by:Catalent, Inc based inSomerset, NEW JERSEY (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 hope for real cures. At Catalent, we understand the unique ...
by:Oligomerix, Inc. based inWhite Plains, NEW YORK (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 diseases shown in Chart A below have a tau etiology similar to AD. Oligomerix’s 1st clinical program ...
Manufactured by:Bausch Health Companies Inc. based inLaval, QUEBEC (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. ...
Manufactured by:Suvoda based inConshohocken, PENNSYLVANIA (USA)
Our advanced patient randomization and trial supply management system. Your clinical trial command and control ...
Manufactured by:Lipella Pharmaceuticals Inc. based inPittsburgh, PENNSYLVANIA (USA)
The following is a link to the Radiation Cystitis Patient Registry. If you are a cancer survivor with a history of pelvic radiation therapy, please consider joining the registry. The potential uses of this registry about radiation cystitis and hemorrhagic cystitis (and cancer survivorship post-radiotherapy) include: improving the scientific understanding of radiation cystitis, discovering trends ...
by:Palvella Therapeutics. based inWayne, PENNSYLVANIA (USA)
QTORIN™ rapamycin 3.9%is a novel targeted topical therapy being studied for Gorlin syndrome. Palvella is enrolling a phase 2b clinical study (CODY) for people with Gorlin ...
by:Cartesian Therapeutics based inGaithersburg, MASSACHUSETTS (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, intellectual disability, and chronic kidney ...
by:Alexion Pharmaceuticals, Inc. based inBoston, MASSACHUSETTS (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 collaboration with leading children’s hospitals and technology and data-science ...
by:Gain Therapeutics, Inc. based inBethesda, MARYLAND (USA)
Mucopolysaccharidosis type 1 (MPS 1) is a rare lysosomal storage disease that is caused by mutations in IDUA, the gene that encodes the alpha-L-iduronidase (IDUA) enzyme. These mutations cause the misfolding and dysfunction of IDUA, which leads to the toxic buildup of large sugars in the bone, cartilage, cornea, heart and central nervous system (CNS). There is currently no cure for these ...
Manufactured by:Pluristem Therapeutics Inc. based inHaifa, ISRAEL
PLX-R18 cells release a combination of therapeutic proteins in response to a damaged or poorly functioning hematopoietic system; this system creates the blood cells that protect us from infection, uncontrolled bleeding and anemia. PLX-R18 is currently in a Phase I clinical trial to treat incomplete hematopoietic recovery following Hematopoietic Cell Transplantation (HCT) and in development to ...
Manufactured by:KORU Medical Systems based inMahwah, NEW JERSEY (USA)
Chronic inflammatory demyelinating polyneuropathy, or CIDP, is a rare disorder characterized by gradually increasing sensory loss and weakness associated with loss of reflexes. This is caused by an autoimmune process in which the body’s own immune system doesn’t recognize its nerve fibers and attacks them. This leads to inflammation of nerve roots and peripheral nerves, as well as ...
Manufactured by:Lupagen Inc. based inDallas, TEXAS (USA)
Lupagen avoids the cost & complexity of cell therapy and the safety & clinical challenges of viral-based gene therapy by a simple patient-connected gene delivery procedure done at the bedside. Our Side CAR-T® delivery system, a first-in-class bedside system enables gene delivery directly to target cells in a highly-controlled and safe environment before reinfusing therapies to the ...
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 liquid taken either orally or through a ...
Manufactured by:InvivoSciences, Inc. (IVS) based inMadison, WISCONSIN (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 skeletal muscle as well as connective tissue, we offer rare disease ...
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. EB affects both genders and every racial and ethnic ...
![Ibio - Model 100 - Idiopathic Pulmonary Fibrosis [IPF] Therapeutics](https://d32zuqhgcrpxli.cloudfront.net/eyJidWNrZXQiOiJlZS1maWxlcyIsImtleSI6ImZpbGVzLzEyNTA4Ny9wcm9kdWN0cy84NTMxMzQvMTI1MDg3XzJfMjAyMzAyMTMwNjEwNTM5ODQxNzI5X3Jhdy5wbmciLCJlZGl0cyI6eyJyZXNpemUiOnsiZml0IjoiY29udGFpbiIsImJhY2tncm91bmQiOnsiciI6MjU1LCJnIjoyNTUsImIiOjI1NSwiYWxwaGEiOjF9LCJ3aWR0aCI6MjQwLCJoZWlnaHQiOjE0NX19fQ== "Ibio - Model 100 - Idiopathic Pulmonary Fibrosis [IPF] Therapeutics")
Manufactured by:Ibio, Inc. based inBryan, TEXAS (USA)
Idiopathic pulmonary fibrosis [IPF] is a form of progressive pulmonary fibrosis, or abnormal scarring of the lungs. As the disease progresses, the increased scarring leads to decreasing transfer of oxygen into the bloodstream, and ultimately, irreversible loss of lung function. The average life expectancy after an IPF diagnosis is 3-5 years.1 Because there is no cure and no therapy has been shown ...
Manufactured by:MedChemExpress LLC (MCE) based inMonmouth Junction, NEW JERSEY (USA)
Maralixibat (SHP625) chloride is an orally active ileal bile acid transporter (IBAT) inhibitor. Maralixibat chloride can be used for the research of rare cholestatic liver diseases including Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC) and biliary ...