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'Not-So-Haunted House,' Bake Sale Raises Over $10,000 For Child With Baby's Alzheimer's

`The “Not-So-Haunted House” and bake sale, which raised money for Damian, a Santa Clarita boy with Acid Sphingomyelinase Deficiency (ASMD), often referred to as “baby’s Alzheimer’s.” raised over $10,000. ....

Santa Clarita , Brittany Markham , Save Damian Campaign , Acid Sphingomyelinase Deficiency , Niemann Pick Disease Types , Save Damian ,

#Save Damian: Santa Clarita 'Baby Alzheimers' Patient Starts Experimental Treatment

#Save Damian: Santa Clarita 'Baby Alzheimers' Patient Starts Experimental Treatment
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Brittany Markham , Santa Clarita , Damian Markham , Wylder Nation Foundation , Acid Sphingomyelinase Deficiency , Niemann Pick Disease Types ,

Save Damian: Santa Clarita Family Raises $800,000 For Their Terminally Diagnosed Child

Ever since their child received a terminal diagnosis at 2-years-old last year, a Santa Clarita family fundraised almost $800,000 for research on the “baby Alzheimer's” disease that will likely take the life of their son before his fifth birthday. ....

Brittany Markham , Santa Clarita , Damian Gofundme , Wylder Nation Foundation , Acid Sphingomyelinase Deficiency , Niemann Pick Disease Types ,

GoFundMe Set Up After Santa Clarita Child Receives Terminal Diagnosis

GoFundMe Set Up After Santa Clarita Child Receives Terminal Diagnosis
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Brittany Markham , Santa Clarita , Damian Markham , Damian Gofundme , A Santa Clarita , Wylder Nation Foundation , Acid Sphingomyelinase Deficiency , Niemann Pick Disease Types , Acid Sphingomyelinase Deficiency , Anta Clarita Child With Fatal Disease , Santa Clarita Gofundme , Anta Clarita Fatal Illness Donations , Anta Clarita Fatal Disease Diagnosis , Santa Clarita Latest News , Santa Clarita News , Scv News , Local News , Santa Clarita , பிரிட்டானி மார்க்கம் , சாந்தா கிளாரிட்டா , வில்தேர் தேசம் அடித்தளம் , நிஎமான்ன் தேர்ந்தெடு நோய் வகைகள் , சுயவிவரம் செய்தி , ஆன்டா கிளாரிட்டா ,

FDA Grants Rare Pediatric Disease Designation to Polaryx Therapeutics' Treatment for Niemann Pick Disease

FDA Grants Rare Pediatric Disease Designation to Polaryx Therapeutics’ Treatment for Niemann Pick Disease
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Rare Daily Staff
The U.S. Food and Drug Administration granted Rare Pediatric Disease and Orphan Drug designations to Polaryx Therapeutics’ PLX-300 for the treatment of Niemann Pick Disease Types A and B.
Niemann Pick Disease (NPD) Types A and B are a group of ultra-rare and fatal pediatric neurodegenerative disorders caused by defects in acid sphingomyelinase, a lysosomal enzyme which plays a pivotal role in the degradation of sphingomyelin. These genetic defects result in increased intracellular accumulation of sphingomyelin leading to severe neurodegeneration, cerebellar ataxia, and dementia. There is no cure for these diseases with patients receiving only supportive care. ....

Hahn Jun Lee , Drug Administration , Rare Pediatric Disease , Orphan Drug , Polaryx Therapeutic , Niemann Pick Disease Types , Pick Disease , Rare Pediatric Disease Priority Review , Eiger Biopharmaceuticals , ஹான் ஜூன் லீ , ரேர் குழந்தை நோய் , ஆர்ஃபந் மருந்து , நிஎமான்ன் தேர்ந்தெடு நோய் வகைகள் , தேர்ந்தெடு நோய் , ரேர் குழந்தை நோய் ப்ரையாரிடீ விமர்சனம் , ஈகர் உயிர் மருந்துகள் ,