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David Law and Veena Sison-Law from Cure GM1 Foundation continue to be advocates and part of a support network for their community, even after their daughter passed. "They embraced all the joys of being her parents — and also carried the pain." It is an inspiring story that you must read... and share.
After years of battling GM1 gangliosidosis, Violet Law died at 4 years old on Saturday, Nov. 11, surrounded by her family.
lbpost.comThis opinion article from The Seattle Times will be a story familiar to many in the rare disease community. Clinical trials showed promise, and patients expected a green light for drug approval. Instead, the U.S. Food and Drug Administration issued a “refusal-to-file” letter and declined to ...examine the trial data. The FDA insists on large trials showing clear evidence of a drug’s clinical effectiveness, meaning that symptoms improve or disappear. But that doesn’t make sense for rare diseases. The agency’s denial was devastating for families, and the FDA needs to reconsider how it evaluates #raredisease drugs.
The federal government should look for ways other than large clinical trials to approve drugs. Patients with rare and ultrarare diseases are losing...
www.seattletimes.comAbbyStrong Fights NPC was diagnosed with #NPC, a terminal genetic metabolic disorder referred to as childhood #dementia. Her family says they remain committed to helping her live a full life. "Tomorrow is never promised to anyone. You just make the best of your life."
An 8-year-old girl from Chesterfield was diagnosed with NPC, terminal childhood dementia. Her family says they remain committed to helping her live a...
www.wral.comThis digital artist’s mission is to empower everyone to pursue their individual talents and gifts to the best of their ability, to demonstrate the power of faith and hard work in the lives of all. Furthermore he is an advocate for the #cerebralpalsy community. #believe ...https://www.instagram.com/reel/C0CP542g1fF/?igshid=MzRlODBiNWFlZA==
Our trips to the grocery store were only the beginning. Judy and I began to analyze every part of our day with our daughter. Each moment became an opportunity to implement therapy sessions and make progress. Creating this lifestyle before gene therapy prepared us extensively for when Rylae-Ann ...would finally be able to make substantial progress after it.
To support his daughter with AADC deficiency, columnist Richard E. Poulin III turned everyday tasks into helpful therapy sessions.
aadcnews.comHere is how to modify an assignment to help students with challenges in writing. Rylae has challenges in gross and fine motor skills. This is how I help her feel successful in the classroom. #modifyinghomework #specialneeds #specialneedsmom #specialneedsteacher #finemotortips #classroomtips
A common theme in the #raredisease community; parents fighting for a chance to have future for their children. Zander's family hopes to have #genetherapy for #leighsyndrome.
Doctors said it's unlikely Zander Marshall would make it to his 3rd birthday.
www.cbsnews.comRylae exploring with colors
With cost being one of the major hurdles in the treatment of #rarediseases, Indian drug companies have started manufacturing medicines for at least four conditions — #Tyrosinemia Type 1, #GaucherDisease, #WilsonDisease and #dravetsyndrome — bringing down the cost by up to 100 fold."
In addition to rare diseases, the government has also urged the industry to produce an oral solution for hydroxyurea needed for the treatment of...
indianexpress.com#Brain reserve was once regarded as having a fixed capacity, but this view has now changed. Not only is there evidence that our brains may be able to make new neurons throughout adulthood, but we now know that many things can influence the functioning of the brain’s hardware. This has led to the ...recognition of a third pillar that underlies cognitive resilience: brain maintenance, which is the lifestyle or environmental factors that keep the brain in good condition.
Why does mental effort lead to a more resilient brain that can withstand dementia and decline? We are now discovering the mechanisms behind this...
www.newscientist.comHomework time with Mommy using clear holder sheet so that she can practice over and over again. #worksheet #reusableworksheet #homework #mathhomework
