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Fourteen-year-old Emily Deacon has a chromosome disorder that is so rare even the specialists don’t have a name for it. She was born with club feet and epilepsy and, as a teenager, still does not speak and is in a wheelchair. When she was first diagnosed, her mum, Liz, said...
To commemorate World Rare Disease Day, the Organisation for Rare Diseases India will hold the 8th edition of ‘Race for Seven’ on Sunday. The event’s objective is to raise awareness of the need for preventative measures to contain rare diseases such as consanguineous marriages, prenatal tests, and informed decisions about...
The Markham family started a GoFundMe page to raise funds to save their son Damian’s life from acid sphingomyelinase deficiency on May 17, 2021. On Feb. 28, the family hit a significant mark on their fundraising journey – raising $1 million. ASMD is a rare progressive genetic disorder that can...
NEW DELHI — Recuperating in the cardiac care unit of Batra Hospital, 20-month-old Stawan Bijal Shah refuses his feed and cries for his mother. He is one of the youngest patients to undergo a coronary artery bypass surgery for a rare disease which damaged his heart. When Stawan was just...
For the first time since 2020, EURORDIS (Rare Diseases Europe) recently held its Black Pearl Awards ceremony in person, recognizing the exceptional work and accomplishments of those who make a difference in the rare disease community.
ABOUT THE VIRTUAL GALA November 6, @ 8 PM EDT Presented by the Kissam Family and Mark & Rachel Rohr Family Foundation. Be inspired by stories from our community, hear from IRSF leadership, learn about the incredible work that our foundation is doing to accelerate research and empower families. We’re especially...
Bloomington, MN – Pandemic-era changes may have permanently altered the clinical trial landscape as decentralized clinical trials gain broader acceptance. During COVID-19, more than three-quarters (76%) of pharmaceutical companies, device manufacturers and contract research organizations conducted off-site clinical trials. These types of trials specifically provide increased opportunity for individuals with...