Genetic Disorders

• PKU from the Mayo Clinic

• PKU from the Cleveland Clinic

• PKU: Kid's Health

When you finish, check out these resources!

• FDA Approves Sepiapterin for Adults, Children Living With Phenylketonuria

• Phenylketonuria and Cognitive Function: Intervening Early Helps, But Long-Term Gaps Remain

• Mayo Clinic: Cystic Fibrosis

• Kids Health: Cystic Fibrosis

• About Cystic Fibrosis

• Cystic Fibrosis

• Brainpop: Cystic Fibrosis
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When you finish, check out these resources!

• Advancing Cystic Fibrosis Treatment with mRNA Therapies

• Clinical trials begin for a first-in-class, inhaled gene therapy for people with cystic fibrosis

• A cystic fibrosis cure for all? Gene editing shows promise

• Johns Hopkins: Huntington's Disease

• Huntington's Disease

• Genetics Home Reference: Huntington's Disease

When you finish, check out these resources!

• Huntington’s Disease Progression May Be Slowed by Regenerative Strategy

• New Huntington’s treatment prevents protein aggregation

• Two Heads Are Better Than One: Combined Physical and Music Therapy for Late-Stage Huntington’s Disease

• SCID: Cleveland Clinic

• SCID: Kid's Health

• SCID: Children's Hospital

When you finish, check out these resources!

• Screening Newborns for Deadly Immune Disease Saves Lives

• Gene therapy delivers lasting immune protection in children with rare disorder

• Experimental gene therapy treatment created at UCLA gives 'bubble girl' a new life