Ultragenyx Patient Advocacy

Duchenne Muscular Dystrophy (DMD) Awareness Week
2023-02-13 08:00 - 2023-02-19 17:00 (America/New_York)
Duchenne Muscular Dystrophy Awareness Week begins on February 13th through the 19th to call national attention to DMD, support patients and families suffering from this rare disease, and raise money for research. DMD is a genetic disorder that cause gradual loss of muscle function and weakness.

Rare Liver Disease Month
2023-02-01 08:00 - 2023-02-28 17:00
During the month of February, the Global Liver Institute will lead the third annual Rare Liver Diseases Month to promote awareness and educate people about the diverse range of liver diseases.

Externally led Patient Focused Drug Development (PFDD) Meeting on Cerebral Creatine Deficiency Syndromes (CCDS)
2023-01-24 10:00 - 15:15
The virtual meeting provides patients and caregivers with an opportunity to share their personal experiences regarding the symptoms and daily impact with CCDS to FDA representatives, academic and scientific researchers, medical professionals, and pharmaceutical companies.

International Day of Persons with Disabilities
2022-12-03
The United Nations’ day of international observance aims to promote an understanding of disability issues and mobilize support for the dignity, rights, and well-being of persons with disabilities. The term disability is used to refer to individual functioning, including physical impairment, sensory impairment, cognitive impairment, intellectual impairment, mental illness, and various types of chronic disease. This observance day also seeks to increase awareness of the importance of integrating persons with disabilities in every aspect of political, social, economic and cultural life.
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FAST's 15th Annual Global Science Summit & Gala
2022-12-02 - 2022-12-03
Foundation for Angelman Syndrome Therapeutics (FAST).
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2022 PCORI Annual Meeting
2022-10-26 08:00 - 2022-10-28 17:00
The 2022 PCORI Annual Meeting will feature keynotes, plenaries, and breakout sessions that highlight a range of timely and important topics around patient-centered research.
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Mitochondrial Disease Awareness Week
2022-09-18 - 2022-09-24
During the third week of September, the Mito community comes together to raise awareness for Mitochondrial Disease Awareness Week.
Thousands of families face the reality of living with mitochondrial disease. During Awareness Week, MitoAction and Mito families do their part to educate others about mitochondrial disease.
Awareness is important year-round but highlighting these issues during Mitochondrial Disease Awareness Week provides a time for people to come together and display the passion and strength of those working to improve the lives of those affected by Mito.

Long Beach Sprint to Save 5K Run and 1 Mile Walk
2022-09-10 09:00 - 13:00 | Long Beach, CA
Join the National MPS Society for the first annual Long Beach Sprint to Save event! This 5K run and 1 mile walk will be held at Shoreline Aquatic Park on September 10.
Learn more and register

Nashville Rock and Roll 5K Run and 1 Mile Walk
2022-08-06 08:00 - 11:00 | Nashville, TN
The Nashville Rock and Roll 5K Run and 1 Mile Walk will take place on Saturday, August 6 as a part of the National MPS Society’s Annual Family Conference! Transportation will be provided to and from the event, which will be held at Centennial Park. Check back for more information regarding departure times.
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National MPS Society 36th Annual Family Conference
2022-08-04 - 2022-08-06
Save the date for the National MPS Society’s 36th Annual Family Conference. More details to come!
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International Metabolic Conference
2022-07-22 08:00 - 2022-07-24 17:00
Join MitoAction and the International Network for Fatty Acid Oxidation Research and Management (INFORM) for the 3rd Annual International Metabolic Conference for Fatty Acid Oxidation Disorders (FAODs) held in Pittsburgh, PA on July 22, 23, & 24, 2022.
In partnership with Dr. Jerry Vockley and INFORM, MitoAction will assemble leading experts to present to families, caregivers, family members and clinicians. The focus of the International Metabolic Conference is for families to learn more about disease management, current clinical trials, nutrition and the social needs of caring for a child or an adult with a rare metabolic disorder. This is a great opportunity for families from across the globe to virtually meet one another, and have the opportunity to interact with professionals who specialize in metabolic disorders.
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The MAGIC Foundation’s 28th Annual Convention
2022-07-14 08:00 - 2022-07-16 17:00 | Lombard, IL, Lombard, IL, United States
Join the MAGIC Foundation for an educational experience for parents of children living with an endocrine disorder. World renowned speakers from across the globe will present on physical health, mental health, insurance, genetics, education issues, and so much more!
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2022 OIF National Conference
2022-07-09 - 2022-07-10
The 2022 OIF National Conference will be held virtually during the weekend of July 9, 2022. More details to come!
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Third Annual Cross Country Challenge Kickoff
2022-06-23
More details coming soon.
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Virtual Capitol Hill Visits with Senators and Representatives
2022-06-23

XLH Awareness Day
2022-06-23
June 23 is X-linked hypophosphatemia (XLH) Awareness Day. Each year, the XLH community works to educate the general public about the condition and celebrate those who are #XLHStrong.
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PPMD Annual Conference
2022-06-22 - 2022-06-26 | Scottsdale, AZ
A comprehensive annual, international conference focused entirely on Duchenne.
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DIA 2022 Global Annual Meeting
2022-06-19 08:00 - 2022-06-23 17:00
The DIA 2022 Global Annual Meeting brings together thousands of innovators from around the globe to discuss updates in the ever-changing field of life sciences.
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Worldwide CDKL5 Awareness Day
2022-06-17
Each June, the CDKL5 community works to educate and raise awareness about the disorder. June has special meaning to the CDKL5 community, and June 17 is Worldwide CDKL5 Awareness Day.
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Spontaneous Parent Group Meeting
2022-06-15

Million Dollar Bike Ride
2022-06-11 07:30 - 17:00 | Philadelphia, PA
The Penn Medicine Orphan Disease Center (ODC) will host the 9th Annual Million Dollar Bike Ride on Saturday, June 11, 2022, to raise money for rare disease research.
Learn more and register

Global Genes 2022 RARE Drug Development Symposium
2022-06-08 - 2022-06-10
The RARE Drug Development Symposium, in partnership with the Orphan Disease Center of the University of Pennsylvania, is designed to connect, educate, and inspire rare disease participants, whether they’re beginning or advanced. This program convenes key stakeholders across patient advocacy, research, and drug development to engage on emerging challenges, and new frameworks to advance rare disease research and therapeutic development.
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Los Angeles Regional Meet-up
2022-06-04

CureDuchenne Futures Conference
2022-05-27 - 2022-05-29 | Orlando, FL
This conference focuses on bringing education, resources, and connection to the Duchenne community in a family-friendly environment.
Learn more and register

International MPS Awareness Day
2022-05-15
Every year on May 15, the mucopolysaccharidosis (MPS) and mucolipidosis (ML) community comes together to recognize those diagnosed, think about the loved ones who have been lost, thank doctors and scientists dedicated to finding cures, and acknowledge parents, siblings, and caregivers who work tirelessly to support those with MPS and ML.

Napa MPS Race for a Cure
2022-05-01 10:00 - 14:00 | Napa, CA
Join the National MPS Society in Napa, California for the 6th Annual Napa MPS Race for a Cure on May 1, 2022. This event will be held at Kennedy Park and will feature a 5K run, 1 mile walk, family picnic, raffle, and silent auction!
Register

Adolescent/Young Adult Group Meeting
2022-04-20

Aging with XLH Group Meeting
2022-04-20

Raleigh Run for Rare
2022-03-26 08:00 - 12:00 | Raleigh, NC
Join the National MPS Society at Dorothea Dix Park on March 26th for the Raleigh Run for Rare! This event will feature a 5K run, 1 mile walk, and post-run family picnic at Harvey Hill.
Register

Advocacy Webinar and Virtual Capitol Hill Registration Kickoff
2022-03-23
