By Dana Talesnik
Isabella and Julia Flysjo, age 4, are part of a first-in-human gene therapy trial at the Clinical Center for their rare disease.
Photo: Niclas Flysjo
It’s agonizing for parents to watch their children suffer and not know how to help them. When parents learn their child has a rare disease, consumed with grief and fear, they try to navigate the confusing medical lingo for diseases that have limited resources and few, if any, effective treatments.
Two families were among those who shared their rare disease journeys during NIH’s Rare Disease Day virtual event on Mar. 1. In both cases, their babies were born seemingly healthy. Then, around age 2, worrisome symptoms appeared that led these families to seek a medical evaluation.
PRA s Center for Rare Diseases Launches Toolkit to Identify and Mitigate Risks to Rare Disease Clinical Programs
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PRA s Center for Rare Diseases Launches Toolkit to Identify and Mitigate Risks to Rare Disease Clinical Programs – Africa News Wire
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Tuberous Sclerosis Alliance. The campaign is underwritten by presenting sponsor Neurelis, Inc. with generous supporting funding from UCB, Inc. SAP Week will highlight the need for people with epilepsy to have a conversation with their healthcare providers, especially if they are in a key subgroup who would benefit from a seizure action plan like those who have intractable epilepsy syndromes, who are at high risk for seizure clusters, status epilepticus or have frequent nocturnal generalized tonic-clonic seizures, said Dr. Tracy Dixon-Salazar, LGS Foundation Executive Director.
SAP Awareness Week features a social media campaign and new website designed to highlight the need for people with epilepsy, their caregivers and healthcare providers to develop detailed plans, especially should an emergency occur. The social media campaign incorporates the hashtag #SAPAW2021 and urges people to learn more at SeizureActionPlans.org.
0 harold ford jr. we have the former government of vermont howard dean and in washington we have the anchor for bbc world news america, katty kay. what a day yesterday. my gosh. we have a lot to talk about. we have politics to talk about, the policy. doctor, you have a lot of concern about it. the democratic uprising in the house and senate that may have led to this. first let s take it down to d.c. with katty. get us up to date on everything that happened yesterday. health care all the time. two headlines do say a lot about the fight over obama care. in the wall street journal, obama retreats. and in usa today, health law shakes the presidency. with democrats threatening to to promote the core values that i think led them to support this thing in the first place. i think it is not possible for me to guarantee that 100% of the people, 100% of the time going on this website will have a perfectly seamless, smooth experience. we re going to have to continue to improve it even after