🚨 #BREAKING: The @fda.gov has approved Denalitx's Tividenofusp alfa-eknm (AVLAYAH)❗

The drug is indicated for treatment of neurologic manifestations of Hunter syndrome (mucopolysaccharidosis type II [ #MPSII ]) in pediatric patients.

#FDA #FDANews #DrugApproval #Neurology #Wow

New in JIMD Reports:
Open-label phase 2/3 substudy of intrathecal idursulfase-IT in children <3 years with neuronopathic MPS II.
Reports long-term follow-up with generally stable cognitive assessments and reduced CSF GAG levels in a small cohort.
🔗 doi.org/10.1002/jmd2...
#MPSII #HunterSyndrome# IMD

www.youtube.com/watch?v=71vG...
Engaging, accessible & informative
7 & 1/2 min video tells the story of precious Oliver, the 1st #MPSII #HunterSyndrome recipient of a groundbreaking #gene-therapy treatment.

From California to Manchester: How One Gene Therapy Is Rewriting Hunter Syndrome Care - Biotech Industry Examiner Hunter syndrome – or mucopolysaccharidosis type II (MPS II) – is the kind of diagnosis that collapses a family’s sense of time.

A 3-year-old from California flies to Manchester for gene therapy.

Our new piece on how rare-disease care is becoming “passport medicine”.
🔗 biotech.industryexaminer.com/passport-med...

#biotech #genetherapy #raredisease #HunterSyndrome #MPSII #GlobalHealth #HealthPolicy #TechNews

Hunter syndrome: Boy with rare condition amazes doctors after world-first gene therapy Oliver has an inherited condition called Hunter syndrome, which causes progressive damage to the body and brain.

#OliverChu has a rare, inherited condition called #Huntersyndrome or #MPSII which causes progressive damage to the body and brain. In the most severe cases, patients with the disease usually die before the age of 20, sometimes described as a type of childhood #dementia.
www.bbc.com/news/article...

Boy with rare condition amazes doctors after world-first gene therapy Oliver has an inherited condition called Hunter syndrome, which causes progressive damage to the body and brain.

#HunterSyndrom #MPSII
#Gene #Therapy 🥰💚✌️
www.yahoo.com/news/article...

Neuroscience Weekly News – September 10th 2025 🧠 This Week in Neuroscience: PTSD Treatment, MS Stem Cell Therapy and More Stay informed with the latest neuroscience breakthroughs! […] The post Neuroscience Weekly News – September 10th 2025 appeared first on LucidQuest Ventures.

LucidQuest Views >>> Neuroscience Weekly News – September 10th 2025 #News #MPSII #biosimilars #multiplesclerosis #PTSD Comment below!

Click Subscribe #Regenxbio #FDA #MPSII #GeneTherapy #Investing

Barbara Burton, MD, Professor of Pediatrics at Northwestern University Feinberg School of Medicine, discusses a recent study examining the effectiveness of idursulfase in young patients with MPS II.

checkrare.com/effectivenes...

#CheckRare #MPSII #RareLysosomal #RareMetabolic

I’m jumping into a freezing cold lake to help give my son a chance to grow up. Support my cause today so we can find a cure for Hunter syndrome.

polarotary.rallyup.com/3e4e24/m/kri...

#HunterSyndrome #MPSII #RareDisease #LakeArrowhead #ProjectAlive #Charity #PolarPlunge