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KRN23 Monoclonal Antibody | TIO | Ultragenyx
http://www.ultragenyx.com/pipeline/krn23-tio
KRN23 is an investigational recombinant fully human monoclonal IgG1 antibody against the phosphaturic hormone Fibroblast Growth Factor 23 (FGF23) in development for tumor-induced osteomalacia (TIO). TIO is characterized by hypophosphatemia, osteomalacia, muscle weakness, fatigue, bone pain, and fractures. Ultragenyx and Kyowa Hakko Kirin entered into a collaboration and license agreement in August 2013 to develop and commercialize KRN23. News and Other Information. Phase 2 Open-Label Adult Study. Emil D&...
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XLH for Patients | Bone Disease | Endocrine | Rare Disease | Ultragenyx
http://www.ultragenyx.com/patients/xlh
What Is X-Linked Hypophosphatemia? A SURVEY OF XLH PATIENTS IS BEING CONDUCTED. CLICK FOR MORE INFO. When the PHEX gene is affected by mutations, too much Fibroblast Growth Factor 23 (FGF23) is made in bone cells. FGF23 blocks phosphate re-absorption by the kidney and suppresses the vitamin D dependent phosphate absorption by the intestine resulting in abnormally low levels of phosphate in the blood. This confidential survey asks you questions about your/your childs experience with XLH, including family ...
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Glut1 DS for Patients | Metabolic Disorder | Seizures | Movement Disorder | Rare Disease | Ultragenyx
http://www.ultragenyx.com/patients/glut1
What is Glut1 DS? Glucose transporter type-1 deficiency syndrome (Glut1 DS) is a rare genetic disorder. The body moves glucose into the brain through a ‘vehicle’ called Glut1 transporter. This Glut1 vehicle is made by a gene called SLC2A1. In people with Glut1 DS, a mutation in this gene means that they cannot make the vehicle properly. Therefore, not enough glucose moves into the brain, which leaves the brain starving for energy. For more information on the study, please visit Glut1Study.com. Ultragenyx...
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GNE Myopathy for Patients | Muscle Disease | HIBM | Rare Disease | Ultragenyx
http://www.ultragenyx.com/patients/gnem
What Is GNE Myopathy? GNE Myopathy (GNEM) is a severe muscle disease that affects adults. A deficiency of an enzyme (GNE/MNK) causes GNE Myopathy. In people with this deficiency, their bodies cannot complete the first step of sialic acid biosynthesis needed for the modification of proteins and fats. GNE Myopathy is also known as Hereditary Inclusion Body Myopathy (HIBM), Distal Myopathy with Rimmed Vacuoles (DMRV) or Nonaka Myopathy. Sephardic Health Organization for Referral and Education (SHORE). Is on...
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Partnering | Business Development | Rare Disease Drug Development and Commercialization | Metabolic | Endocrine | Neurology | Ultragenyx
http://www.ultragenyx.com/partnering
Since 2010, we have built our pipeline of rare disease products largely based on collaborations and licenses with both academic and corporate partners. We continue to seek new opportunities at all stages of development. Parties developing products or technologies for rare genetic diseases that are interested in collaborating with Ultragenyx can contact our business development team. Send any inquiries by email to:. Emil D. Kakkis, M.D., Ph.D. Jayson Dallas, M.D. Daniel G. Welch. Matthew K. Fust.
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Dedicated to Patients & Caregivers | Rare Diseases | Ultragenyx
http://www.ultragenyx.com/patients
Our Commitment to Patients. Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company working to develop novel products for the treatment of rare and ultra-rare diseases. Our focus is on improving the lives of patients who have no approved therapies for their diseases. Everyone in our company shares the same goal: delivering safe and effective therapies as quickly as possible to the people who need them. About Clinical Studies for Rare Diseases. PHASE 1/2 OR 2. People with the disease. Ultragenyx is ...
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TIO for Patients | Bone Disease | Endocrine | Rare Disease | Ultragenyx
http://www.ultragenyx.com/patients/tio
What is Tumor-Induced Osteomalacia? Tumor-induced osteomalacia (TIO) is caused by slow-growing tumors that overexpress a protein called Fibroblast Growth Factor 23 (FGF23). FGF23 is involved in phosphate absorption, which plays an important role in bone health. A variant of TIO called epidermal nevus syndrome (ENS) is associated with skin lesions and results in similar symptoms. The diagnosis is confirmed by a blood test for FGF23 and locating the tumor through imaging. Jayson Dallas, M.D. Clay B. Si...
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Internships | Careers | Rare Disease Drug Development and Commercialization | Ultragenyx
http://www.ultragenyx.com/careers/internships
The summer internship is a 10 to 12 week program for all eligible students interested in the field of science/biotechnology. Ultragenyx hosts interns for all interested groups across the company. Working as a member of a project team in research, commercial, clinical, regulatory, or a business area, interns are given clearly defined responsibilities and project scopes. Interns are paid a competitive hourly rate, unless credits can be applied towards degree. Students will be responsible for providing ...
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FAOD for Patients | Metabolic Disorder | Rare Disease | Ultragenyx
http://www.ultragenyx.com/patients/faod
What are Long-Chain Fatty Acid Oxidation Disorders? Long-chain fatty acid oxidation disorders (FAOD) are a group of autosomal recessive conditions that prevent the body from breaking down long-chain fatty acids into energy. Beginning from early in life to as late as teenage or adulthood, patients with FAOD present with acute crises of energy metabolism and severe energy deficiency. This can lead to potentially severe complications:. Rhabdomyolysis (breakdown of muscle fibers into the blood). UX007 (trihe...
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Careers | Rare Disease Drug Development and Commercialization | Ultragenyx
http://www.ultragenyx.com/careers
Ultragenyx is committed to creating and maintaining an environment that values hard work, fosters creativity, and promotes success. We have a professional work environment, fueled by the talent and expertise of our employees and driven by our commitment to treating patients with significant unmet medical needs. Our company culture statement is clear and simple – and so ‘us’:. To be a hero for our patients. At Ultragenyx, being a hero means embodying a set of core attributes that define who we are and wha...
