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Rare Conversations: Sheri & Stephanie
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Rare Conversations: Kendra & Preston
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Overview on Understanding Mitochondria?
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Sandra’s Journey in ASMD (Adult Patient)
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Patient Caregiver’s Journey in ASMD
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Phenylketonuria (PKU)
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Testing Angelman and Fragile X Syndrome Therapies in Younger Patients
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OV101 Being Studied in Angelman and Fragile X Syndromes
3598 views | 8 years ago
Glioblastoma survivor shares his story
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Jack’s ASMD Story: Living with Niemann-Pick B
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Hereditary Xerocytosis Overview
3334 views | 8 years ago
22q11.2 Deletion Syndrome
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News
Belite Bio Announces Positive Topline Results from the Pivotal Global,
Cullinan Therapeutics Receives FDA Fast Track Designation for CLN-049,
Solid Biosciences Inc.’s SGT-212 Receives Rare Pediatric Disease Des
Biodexa Announces Enrollment of First European Patients into Pivotal P
Ensoma Announces First Patient Dosed in Phase 1/2 Clinical Trial of EN
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