Special Publications

July 12, 2020 | Ultragenyx 3 Two Approvals in June 2020 Alone Four Approvals in Under Three Years, All for Rare Diseases with No Other Approved Treatment OpĖons Get to Know Some of Our PaGents! Lauren Lauren is currently 15 years old and was diagnosed with XLH when she was born. Because the disease was caught early, she was able to start treatment right away so the condition would not worsen. Although Lauren was on treatment soon after being diagnosed with XLH, running was painful for her and XLH impacted her growth. When she was in 6th grade, Lauren had her first surgery where plates were inserted to try to fix the bowing in her legs. When Lauren was in 9th grade, she had bone removed from her left leg that was replaced with a large plate. The surgeries did not provide as much correction as Lauren and her family were hoping for. Lauren began taking Crysvita® (burosumab) after it was approved in 2018. MaỂhew Matthew was diagnosed with MPS VII when he was 17 months old and he is now 18 years old. Since his diagnosis, he has resided in St. Mary's Hospital for Children in Queens. Matthew was able to spend a small amount of time outside the hospital with his family during the holidays until October 2013; since then he cannot leave the hospital because of respiratory complications. When Matthew was a toddler, his mother approached Dr. Emil Kakkis, our President and CEO, to develop a treatment for Matthew. Ultragenyx began developing an investigational treatment for MPS VII and treated Matthew on a compassionate use basis starting in 2013. Matthew later became the first patient in the first clinical trial of Mepsevii® (vestronidase alfa) and has been on therapy for over 6 years. Matthew is loving and sociable with an infectious smile, and enjoys spending time with family and friends. Sources: www.globalgenes.org/rare-facts (last visited July 2, 2020) and https://rarediseases.info. nih.gov/diseases/pages/31/faqs-about-rare-dis- eases (last visited July 2, 2020) Rare Disease Facts • There are 7,000+ rare disease types • Only 5% of rare diseases have an FDA-approved drug treatment • 80% of rare diseases are caused by genetic defects • Rare diseases affect over 400 million people worldwide; approximately 50% of people affected by rare diseases are children • It is estimated that 25-30 million Americans (almost 1 in 10) have rare diseases • 30% of children with a rare disease will not live to see their 5th birthday • On average, it takes most rare disease patients ~5 years to receive an accurate diagnosis; within this time period, they will have seen over 10 specialists and been misdiagnosed 3 times • Rare diseases impact more people than cancer and AIDS combined

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