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Rare Disease Day 2017 With A Commitment To Provide New Insights Into Rare Diseases Equipment & Supplies
46 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:Healx Ltd. based inCambridge, 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 ...
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:Evox Therapeutics Limited based inOxford, UNITED KINGDOM
Niemann-Pick C disease (NPC) is a lysosomal storage disorder which affects the brain and multiple other ...
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: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 ...
Manufactured by:CENTOGENE N.V. based inRostock, GERMANY
The CentoPharma platform provides intelligent access to our comprehensive data repository that combines phenotype, genotype and biochemistry information with epidemiological data and clinical information from a global cohort originating from more than 120 countries. Dynamic search criteria enable the targeted compilation of individual customized cohorts based on desired geographical region, ...
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 ...
by:Healx Ltd. based inCambridge, 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 ...
Distributed by:Salehiyah Establishment based inRiyadh, SAUDI ARABIA
Explore our diversified portfolio of pharmaceuticals, as we deliver the right medication to the right patient at the right time, covering some of the most critical needs of patients in the Kingdom. ...
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 ...
Manufactured by:Diaceutics PLC based inBelfast, UNITED KINGDOM
What is DXRX Signal? DXRX Signal delivers an actionable alert of a testing behavior that allows you to engage a physician whilst they are making treatment decisions, therefore maximizing the effectiveness of your brand sales and ...
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 ...
Manufactured by:BOSANA Medizintechnik GmbH based inDorsten, 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 Institute of Neuroinformatics (Neural Plasticity Lab) at the well-known ...
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 ...
Manufactured by:Radiopharm Theranostics based inCarlton, AUSTRALIA
DUNP19 is the invention of Professor Ulmert at UCLA. ...
Manufactured by:diaserve Laboratories GmbH based inIffeldorf, GERMANY
Human products are frozen liquid materials based on voluntary blood donations. Materials are intended to support the industries to manufacture their own controls and calibrators. All listed products are selected and processed according to customer's specific ...