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Rare Disease Day 2017 With A Commitment To Provide New Insights Into Rare Diseases Services In Europe
16 services found
Manufactured by:Novo Nordisk A/S based inBagsværd, DENMARK
Drug development works best as a team endeavor. We continuously explore strategic collaborations to advance our pipeline toward commercialization and to further our work in the treatment of both rare diseases and common diseases that have a genetic component. We are eager to discuss collaborative partnerships utilizing our proprietary RNAi ...
by:Genome Biologics based inFrankfurt am Main, GERMANY
Kearns-Sayre Syndrome (KSS) is a mitochondrial disease, with Rare Disease designation (7,400 – 22,000 patients in Europe), caused primarily by large-scale mitochondrial DNA deletions. Cardiac dysfunction is the principal clinical manifestation and primary cause of death. There are no current treatment options available that go beyond addressing some of the symptoms associated with KSS thus ...
by:SeqOne S.A.S. based inMontpellier, FRANCE
We offer a comprehensive solution for hereditary and rare disease data that detects both commonly accessible variants and relevant challenging variants such as mid-sized deletions, Alu elements and larger structural ...
Manufactured by:CENTOGENE N.V. based inRostock, GERMANY
Whole Exome Sequencing (WES) is a comprehensive genetic test that identifies changes in a patient's DNA that are causative or related to their medical concerns. By focusing on the entire protein-coding regions of the genome – the exome – WES offers you the coverage you need to diagnose patients rapidly and ...
Manufactured by:Sangamo Therapeutics based inBrisbane, CALIFORNIA (USA)
Sangamo is using groundbreaking science and novel technology to replace today’s symptomatic treatments with tomorrow’s genomic cures. We are pursuing programs across the spectrum of genomic medicine, with gene and cell therapies in the clinic today and preclinical programs in genome engineering and off-the-shelf cell therapy. Behind these are research efforts thoughtfully focused on ...
by:JADBio based inLos Angeles, CALIFORNIA (USA)
AutoML brings democratization to the data analysis process by allowing everyone to utilize Machine Learning. Fully automate your data analysis, end-to-end, with AI and our purpose-built algorithms starting from preprocessing to interpreting the final ...
Manufactured by:CENTOGENE N.V. based inRostock, GERMANY
We provide a broad range of services to successfully operate clinical trials. We leverage our proprietary Bio/Databank, multiomic platform, extensive physician network and our experts to ensure efficient and effective solutions to drive progress in the rare disease ...
Manufactured by:MorphoSys AG based inPlanegg, GERMANY
Expanded Access to our Investigational Medicinal Products Policy. At MorphoSys, our mission is to make innovative biopharmaceuticals to improve the lives of patients suffering from serious diseases. Before these and all other investigational medicinal products can be made broadly available, the U.S. Food and Drug Administration (FDA) and other health authorities around the world require that ...
by:Azafaros based inNaarden, NETHERLANDS
Lysosomal storage diseases (LSDs) are a group of over 70 diseases that are characterized by lysosomal dysfunction, most of which are inherited as autosomal recessive traits. These disorders are individually rare but collectively affect 1 in 5,000 live births. They typically present in infancy and childhood, although adult-onset forms also occur. Most LSDs have a progressive neurodegenerative ...
Manufactured by:Neovii Pharmaceuticals AG based inRapperswil, SWITZERLAND
Aplastic anemia is a rare, but serious hematological disease caused by a failure or deficiency of the bone marrow to produce blood cells (erythrocytes, leukocytes, and platelets). Patients with aplastic anemia are at risk of life-threatening infections or fatal bleeding. Curative treatments for aplastic anemia include immunosuppressive therapy, and/or hematopoietic stem cell ...
Manufactured by:CENTOGENE N.V. based inRostock, GERMANY
We work with partners to develop new disease models and new biomarkers of disease. We can also use our disease models to identify new drug targets and screen chemical compounds to identify potential new drug candidates. CENTOGENE provides companies with tools to accelerate drug discovery. From population screening studies that elucidate the prevalence of a rare disease to disease modeling, we are ...
by:Azafaros based inNaarden, NETHERLANDS
Azafaros B.V. is pioneering the development of innovative oral small molecule compounds addressing the pathological effects of metabolite accumulation in rare metabolic disorders. Their lead compound, nizubaglustat, leverages unique dual-mode action, making it a promising candidate for modifying diseases irrespective of patient genotypes. Notably effective against conditions such as GM1 and GM2 ...
by:ICON plc based inDublin, IRELAND
Accelerating study start-up and delivering results with custom strategies and logistics solutions. ICON helps navigate the Cell and Gene Therapy journey by overcoming challenges and mitigating risks. Almost every cell and gene therapy clinical trial is novel in some way. Working with living therapies presents new and unique challenges to the traditional clinical trial model. With over 400 ...
Manufactured by:Pluristem Therapeutics Inc. based inHaifa, ISRAEL
Acute Radiation Syndrome (ARS) involves severe, potentially lethal damage to the bone marrow’s ability to produce blood cells, as well as to other systems and organs. Severe damage to bone marrow makes victims vulnerable to life-threatening hemorrhage, infection and anemia. ...
by:ICON plc based inDublin, IRELAND
ICON's cardiovascular trial experience, supported by a global team of experts. ICON has extensive experience in cardiovascular clinical research trials across the entire drug development spectrum. There is still significant unmet need within select cardiovascular/metabolic (CVM) disorders. Unfortunately, the cost of developing drugs and devices in this area has been rising steadily. ICON, a full ...
Manufactured by:Pluristem Therapeutics Inc. based inHaifa, ISRAEL
Bone marrow deficiency refers to a condition in which the hematopoietic stem cells in the bone marrow fail to produce enough, or produce abnormal, red blood cells, white blood cells and platelets. Hematopoietic cell transplantation (HCT), the transplantation of multipotent hematopoietic cells, is a standard treatment in various malignant and non-malignant conditions. Transplant patients are ...