A Cedar Park mother and Texas Original Compassionate Cultivation are working to encourage legislators to remove restrictions on qualifying conditions and more. CEDAR PARK, Texas — Texas lawmakers are back in session and some of the bills headed their way have to do with medical marijuana reform. A Cedar Park...
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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...
A foreign drugmaker has thrown a gauntlet to the Korean rare disease treatment market. Italy-based Recordati is the company. On Rare Disease Day Feb. 28, the company launched Recordati Korea, its Korean offshoot and Asia-Pacific headquarters, declaring its official advance to the Korean and regional market. Unlike most other pharma...
Northwestern University researchers have developed a new injectable therapy that harnesses “dancing molecules” to reverse paralysis and repair tissue after severe spinal cord injuries. In a new study, researchers administered a single injection to tissues surrounding the spinal cords of paralyzed mice. Just four weeks later, the animals regained the...
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...
EURORDIS-Rare Diseases Europe, in collaboration with Rare Diseases International and the NGO Committee for Rare Diseases, has developed a toolkit to support the call for a UN Resolution on Addressing the Challenges of Persons Living with a Rare Disease and their Families. Vulnerability, exclusion and inequity are the reason why...
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...