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Ultragenyx—Treatment of Rare and Ultrarare Genetic Diseases

Ultragenyx is focused on developing first-ever approved treatments for rare and ultrarare diseases and moving the rare disease community forward.
Categories: Business, Health and Wellness
Topics: rare genetic diseases, ultragenyx

Moz DA: 44 Moz Rank: 4.3 Semrush Rank: 194,801
ultragenyx.com
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Sites similar to ultragenyx.com - Top 40 ultragenyx.com alternatives

ultrarareadvocacy.com

ultrarareadvocacy.com ultrarareadvocacy.com           
Ultragenyx Patient Advocacy - Rare and UltraRare Diseases
At Ultragenyx, we strive to incorporate patient & family voices into everything we do. We're passionate about supporting those with rare & ultra-rare diseases.

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xlhnetwork.org

xlhnetwork.org xlhnetwork.org           
Home :: XLH Network
Home :: XLH Network

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crysvita.com

crysvita.com crysvita.com           
CRYSVITA (burosumab-twza) – Official Site for Patients
CRYSVITA (burosumab-twza) is the only FDA-approved treatment for X-linked hypophosphatemia (XLH), a disease that can lead bones to “soften” and weaken over time.

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xlhlink.com

xlhlink.com xlhlink.com           
Soft and weak bones? Have you heard of X-linked hypophosphatemia (XLH)? Learn more about this disease and its symptoms.
XLHLink is dedicated to providing education and support services for XLH (X-linked hypophosphatemia). Learn about the symptoms of and disease management for this rare skeletal disorder.

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kyowa-kirin.com

kyowa-kirin.com kyowa-kirin.com           
Kyowa Kirin
The Kyowa Kirin Group is a Japanese pharma group that develops innovative specialty drugs, to raise the health and well-being of people around the world. Its flagship business Kyowa Kirin Co., Ltd. creates antibody-based drugs for cancer, kidney disease, and immune system diseases.

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g1dfoundation.org

g1dfoundation.org g1dfoundation.org           
Glut1 Deficiency Foundation
Glut1 Deficiency Foundation - help and hope for the Glut1 Deficiency community

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ultracaresupport.com

ultracaresupport.com ultracaresupport.com           
UltraCare
UltraCare by Ultragenyx connects people living with rare diseases to support resources and financial assistance programs needed to manage their treatment journey.


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xlhnewstoday.com

xlhnewstoday.com xlhnewstoday.com           
XLH News Today Home - XLH News Today
The Web's Daily Resource for XLH News Today

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dimensiontx.com dimensiontx.com           

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glut1dsinfocus.com

glut1dsinfocus.com glut1dsinfocus.com           
Glut1 Deficiency Foundation
Glut1 Deficiency Foundation - help and hope for the Glut1 Deficiency community


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xlhalliance.org

xlhalliance.org xlhalliance.org           
International XLH Alliance
We are an alliance of patient groups for individuals affected by x-linked hypophosphatemia and related disorders.

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mepsevii.com

mepsevii.com mepsevii.com           
MEPSEVII™ (vestronidase alfa-vjbk) is the first and only enzyme replacement therapy for MPS VII
Learn about MEPSEVII™ (vestronidase alfa-vjbk)—the first and only enzyme replacement therapy for mucopolysaccharidosis (MPS VII, Sly syndrome).

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xlhlink.eu

xlhlink.eu xlhlink.eu           
Welcome to XLH link
A website focused on sharing the real-life experiences of people with XLH,
plus expert information and new ideas

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healthcap.eu

healthcap.eu healthcap.eu           
Start - HealthCap
HealthCap, a life science investor and company creator. Our motivation is to find innovative ideas and invest that could impact healthcare and society.

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kyowakirin.com

kyowakirin.com kyowakirin.com           
Kyowa Kirin
The Kyowa Kirin Group is a Japanese pharma group that develops innovative specialty drugs, to raise the health and well-being of people around the world. Its flagship business Kyowa Kirin Co., Ltd. creates antibody-based drugs for cancer, kidney disease, and immune system diseases.

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mpssociety.org

mpssociety.org mpssociety.org           
National MPS Society - We Help Cure, Support, & Advocate for MPS & ML
We support life-saving research for mucopolysaccharidosis (MPS) & mucolipidosis (ML), and we provide community resources, support, & more.

Moz DA: 52 Moz Rank: 4.6 Semrush Rank: 344,755 Facebook ♡: 1,906
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everylifefoundation.org

everylifefoundation.org everylifefoundation.org           
EveryLife Foundation for Rare Diseases
We empower the rare disease patient community to advocate for impactful, science-driven legislation and policy that advances the equitable development of and access to lifesaving diagnoses, treatments, and cures.

Moz DA: 46 Moz Rank: 4.6 Semrush Rank: 475,904
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ijmbs.org ijmbs.org           

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xlhlinkhcp.com

xlhlinkhcp.com xlhlinkhcp.com           
Welcome to XLH Link
X-linked hypophosphatemia (XLH)


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sarepta.com sarepta.com           
Sarepta Therapeutics | Biopharmaceutical Company for Rare Diseases
We are focused on the development of precision genetic medicines to treat rare neuromuscular and central nervous system diseases. Learn more about Sarepta.

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mpsviiinfocus.com

mpsviiinfocus.com mpsviiinfocus.com           
MPS 7 - Mucopolysaccharidosis VII | MPS VII In Focus
MPS VII is one of the rarest types of mucopolysaccharidoses2-4
Mucopolysaccharidosis VII (MPS VII), also called Sly syndrome, is a genetic metabolic disorder caused by a deficiency of the lysosomal enzyme β-glucuronidase.1,2
MPS VII is a heterogeneous and progressive disease. It requires early diagnosis for

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thasso.com

thasso.com thasso.com           
thasso
glocalized social network in theragenomic and personalized medicine and individualized drug safety for patients of identical genotype, phenotype, or therapy option

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Pappas Capital
Pappas Capital

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The XLH Network, Inc.

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Fatty Oxidation Disorders Family Support Group - All In This Together
Fatty Oxidation Disorders Family Support Group - All In This Together

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milestonesforchildren.org milestonesforchildren.org           
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Help Milestones for Children fund research for Glucose Transporter Deficiency Syndrome (Glut1 DS), a pediatric brain energy metabolic syndrome. Children with this disorder have a myriad of physical and mental disabilities, ranging from mild to so severe that they cannot walk or talk. Glut1 DS was first discovered in 1991 by Dr. Darryl DeVivo at the Colleen Giblin Laboratories, Columbia Presbyterian Medical Center.

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Cure GSD - CureGSD
The Children's Fund for Glycogen Storage Disease Research is a 501(c)3 not-for-profit foundation that aims to make a difference in the lives of children and families living with GSD1.


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Searchable abstracts of presentations at key conferences on calcified tissues. It is also a permanent, citable record, freely available to all without access restrictions.

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softbones.org

softbones.org softbones.org           
SoftBones :: Home
Home | Hypophosphatasia (hī-pō-ˌfäs-fə-ˈtā-zh(ē-)ə) or HPP is an inherited (genetic) metabolic disorder. People with this condition have low levels of the enzyme alkaline phosphatase (ALP), which often affects the development of bones and teeth.

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Home - PANTHERx Rare

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Learn about the different types of long-chain fatty acid oxidation disorders (LC-FAOD), a group of rare, genetic metabolic disorders.

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priorityreviewvoucher.org priorityreviewvoucher.org           
Home - Priority Review Vouchers
The US Congress created the priority review voucher program in 2007 based on a 2006 Health Affairs paper (Ridley et al. 2006). The voucher entitles the bearer to regulatory review in about six months rather than the standard ten months. … Continued


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The Society for Mucopolysaccharide Diseases (MPS Society) provides professional support to individuals and families affected by MPS, Fabry and related lysosomal storage diseases. Contact our helpline Monday-Friday 9-5pm on 0345 389 9901 or email us at [email protected]


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