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Rare Genetic Diseases | Ultragenyx

Ultragenyx is a clinical-stage biopharmaceutical company committed to bringing to market novel products for the treatment of rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. Founded in 2010, the company has rapidly built a diverse portfolio of product candidates with the potential to address diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are no approved therapies.

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Rare Genetic Diseases | Ultragenyx | ultragenyx.com Reviews

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Ultragenyx is a clinical-stage biopharmaceutical company committed to bringing to market novel products for the treatment of rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. Founded in 2010, the company has rapidly built a diverse portfolio of product candidates with the potential to address diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are no approved therapies.

<META> KEYWORDS

1 transforming good science

2 into great medicine

3 management

4 dennis huang

5 thomas kassberg

6 john pinion

7 shalini sharp

8 board of directors

9 william aliski

10 michael narachi

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transforming good science,into great medicine,management,dennis huang,thomas kassberg,john pinion,shalini sharp,board of directors,william aliski,michael narachi,pipeline,krn23 for xlh,krn23 for tio,rhgus,rhppca,triheptanoin for faod,aceneuramic acid

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Rare Genetic Diseases | Ultragenyx | ultragenyx.com Reviews

https://ultragenyx.com

Ultragenyx is a clinical-stage biopharmaceutical company committed to bringing to market novel products for the treatment of rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. Founded in 2010, the company has rapidly built a diverse portfolio of product candidates with the potential to address diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are no approved therapies.

INTERNAL PAGES

ultragenyx.com

1

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...

2

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 ...

3

UX007 | Substrate Replacement | Synthetic Triglyceride | Glut1 DS | Ultragenyx

http://www.ultragenyx.com/pipeline/triheptanoin-glut1

UX007 for Glut1 DS. UX007 (triheptanoin) is an investigational pharmaceutical-grade, specially designed synthetic triglyceride compound, created via a multi-step chemical process and purified, which is in development for glucose transporter type 1 deficiency syndrome (Glut1 DS). Ultragenyx has licensed rights to UX007 from Baylor Research Institute and UniQuest. UX007 is metabolized into heptanoate, which is intended to diffuse across the blood-brain barrier and provide energy to the brain. Heptanoat...

4

UX007 | Substrate Replacement | Synthetic Triglyceride | FAOD | Ultragenyx

http://www.ultragenyx.com/pipeline/triheptanoin-faod

UX007 (triheptanoin) is an investigational pharmaceutical-grade, specially designed synthetic triglyceride compound, created via a multi-step chemical process and purified, which is in development for long-chain fatty acid oxidation disorders (LC-FAOD). Ultragenyx has licensed rights to UX007 from Baylor Research Institute. News and Other Information. Ultragenyx Announces Positive Interim Data From Phase 2 Study of UX007 in Long-Chain Fatty Acid Oxidation Disorder Patients. Phase 2 Open-Label Study.

5

Product Pipeline | Rare Disease Drug Development | Ultragenyx

http://www.ultragenyx.com/pipeline

Ultragenyx is a clinical-stage biopharmaceutical company committed to bringing to market novel products for the treatment of rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. Founded in 2010, the company has rapidly built a diverse portfolio of product candidates with the potential to address diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are no approved therapies. Mucopolysaccharidosis 7 (MPS 7).

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xlhnetwork.blogspot.com

XLH Network: Kyowa Shares KRN23 Data From a First-in-Human, Single Dose Phase 1 Study

http://xlhnetwork.blogspot.com/2013/10/kyowa-shares-krn23-data-from-first-in.html

Wednesday, October 23, 2013. Kyowa Shares KRN23 Data From a First-in-Human, Single Dose Phase 1 Study. Kyowa Shares KRN23 Data. Announcement of Results from a Single Dose Phase 1 Study of a Human Monoclonal Anti-FGF23 Antibody (KRN23) in X-linked Hypophosphatemia in Adults. About KRN23 and FGF23. About X-linked Hypophosphatemia (XLH). About Ultragenyx Pharmaceutical Inc. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragen...

xlhnetwork.blogspot.com

XLH Network: October 2013

http://xlhnetwork.blogspot.com/2013_10_01_archive.html

Wednesday, October 23, 2013. Kyowa Shares KRN23 Data From a First-in-Human, Single Dose Phase 1 Study. Kyowa Shares KRN23 Data. Announcement of Results from a Single Dose Phase 1 Study of a Human Monoclonal Anti-FGF23 Antibody (KRN23) in X-linked Hypophosphatemia in Adults. About KRN23 and FGF23. About X-linked Hypophosphatemia (XLH). About Ultragenyx Pharmaceutical Inc. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragen...

hkbio.org.hk

ICBEM - International Conference on Brain Energy Metabolism

http://www.hkbio.org.hk/./icbem

Norwegian University of Science and Technology, Norway. Ken Kin Lam Yung,. Hong Kong Baptist University, Hong Kong. University of Alabama Birmingham, USA. Ecole Polytechnique Federale de Lausanne, Switzerland. University of Copenhagen, Denmark. University of Lausanne, Switzerland. Brian MacVicar, The University of British Columbia,. Bruce Ransom, University of Washington,. Lasse Kristoffer Bak, University of Copenhagen,. University of Leipzig, Germany. Peking University, China. L Felipe Barros,. Dear Fri...

thexlhnetwork.blogspot.com

The XLH Network, Inc.: February 2015

http://thexlhnetwork.blogspot.com/2015_02_01_archive.html

The XLH Network, Inc. Tuesday, February 24, 2015. Rare Disease Day (February 28) is mostly about bringing attention to all of the diseases and their impacts. New this year is a related fundraising day, Give RARE Day, on March 3. The event was designed to acknowledge that more people are directly affected by a rare disease, more than the number of cancer patients who get more attention and research dollars. To set up your own crowdsource-fundraiser connected to Give RARE Day, just go to our page, and you'...

thexlhnetwork.blogspot.com

The XLH Network, Inc.: March 2015

http://thexlhnetwork.blogspot.com/2015_03_01_archive.html

The XLH Network, Inc. Tuesday, March 31, 2015. West Coast XLH Day registration is closed. West Coast XLH Day registration is now closed. We hope you've already secured your spot, and we look forward to meeting new friends and catching up with old friends. Monday, March 30, 2015. Last chance for XLH Day registration. Wednesday, March 25, 2015. XLH Days, past, present and future. In the course of planning the West Coast XLH Day (registration is still open at xlhday.com. If you've got a story from a prior e...

dgm-kongress.de

23. Kongress des Wissenschaftlichen Beirates der Deutschen Gesellschaft für Muskelkranke e.V. (DGM) 16.-17. Februar 2017 - Künstlerhaus München: Anreise

http://www.dgm-kongress.de/anreise-hotels/anreise

23 Kongress des Wissenschaftlichen Beirates der Deutschen Gesellschaft für Muskelkranke e.V. (DGM). 16-17 Februar 2017 - Therapie neuromuskulärer Erkrankungen im Künstlerhaus, München. 18 Februar 2017 - Patiententag der DGM e.V. in der Pfennigparade Rehabilitationszentrum, München. 23 Kongress des Wissenschaftlichen Beirates der Deutschen Gesellschaft für Muskelkranke e.V. (DGM). 16-17 Februar 2017 - Therapie neuromuskulärer Erkrankungen im Künstlerhaus, München. Finden Sie alle relevanten Informationen,...

dgm-kongress.de

23. Kongress des Wissenschaftlichen Beirates der Deutschen Gesellschaft für Muskelkranke e.V. (DGM) 16.-17. Februar 2017 - Künstlerhaus München: Kontakt

http://www.dgm-kongress.de/kontakt

23 Kongress des Wissenschaftlichen Beirates der Deutschen Gesellschaft für Muskelkranke e.V. (DGM). 16-17 Februar 2017 - Therapie neuromuskulärer Erkrankungen im Künstlerhaus, München. 18 Februar 2017 - Patiententag der DGM e.V. in der Pfennigparade Rehabilitationszentrum, München. 23 Kongress des Wissenschaftlichen Beirates der Deutschen Gesellschaft für Muskelkranke e.V. (DGM). 16-17 Februar 2017 - Therapie neuromuskulärer Erkrankungen im Künstlerhaus, München. Telefon 49 3641 3116-374.

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23. Kongress des Wissenschaftlichen Beirates der Deutschen Gesellschaft für Muskelkranke e.V. (DGM) 16.-17. Februar 2017 - Künstlerhaus München: Medienkooperationen

http://www.dgm-kongress.de/aussteller-sponsoren/medienkooperationen

23 Kongress des Wissenschaftlichen Beirates der Deutschen Gesellschaft für Muskelkranke e.V. (DGM). 16-17 Februar 2017 - Therapie neuromuskulärer Erkrankungen im Künstlerhaus, München. 18 Februar 2017 - Patiententag der DGM e.V. in der Pfennigparade Rehabilitationszentrum, München. 23 Kongress des Wissenschaftlichen Beirates der Deutschen Gesellschaft für Muskelkranke e.V. (DGM). 16-17 Februar 2017 - Therapie neuromuskulärer Erkrankungen im Künstlerhaus, München.

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Rare Genetic Diseases | Ultragenyx

For rare genetic diseases. Deep and diversified product pipeline. Developing multiple clinical-stage programs in parallel. Focused on serious rare and ultra-rare diseases. Working with patients and medical professionals to treat rare diseases. Dedicated to creating new treatments. Join a high-performance team focused on rare diseases. Aug 13, 2015. Ultragenyx Reports Second Quarter 2015 Financial Results and Corporate Update. Aug 6, 2015. Aug 5, 2015. Emil D. Kakkis, M.D., Ph.D. Sunil Agarwal, M.D.

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