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Rare Conversations: Kendra & Preston
9905 views | 6 years ago
Rare Conversations: Sheri & Stephanie
9879 views | 6 years ago
Overview on Understanding Mitochondria?
9851 views | 6 years ago
Patient Caregiver’s Journey in ASMD
8461 views | 6 years ago
Sandra’s Journey in ASMD (Adult Patient)
8292 views | 6 years ago
Phenylketonuria (PKU)
3896 views | 6 years ago
Testing Angelman and Fragile X Syndrome Therapies in Younger Patients
3460 views | 6 years ago
OV101 Being Studied in Angelman and Fragile X Syndromes
3325 views | 6 years ago
Glioblastoma survivor shares his story
3269 views | 6 years ago
Jack’s ASMD Story: Living with Niemann-Pick B
3211 views | 6 years ago
Hereditary Xerocytosis Overview
3075 views | 6 years ago
22q11.2 Deletion Syndrome
2705 views | 6 years ago
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News
First Clinical Trial Results of Gene Therapy (GNT0004) for Duchenne Mu
Chiesi Global Rare Diseases Announces Publication of Results from Fabr
Edgewise Receives European Medicines Agency (EMA) Orphan Drug Designat
Calliditas Announces Positive NefIgArd Open Label Extension Results in
Neurocrine Biosciences Launches WHAT THE C@H?! Educational Initiative
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