New Alagille syndrome therapy named ART4 gets FDA designations
The U.S. Food and Drug Administration (FDA) has granted orphan drug and rare pediatric disease designations to ART4, an experimental therapy for Alagille syndrome…
Alagille syndrome
Facing unexpected complications after a routine surgery
The last few weeks have been a roller coaster as we near the end of summer, prepare for school to start, and navigate a surgery…
New battles royales as new medicines are added to the mix
My son Finley is like most other kids his age: He benefits from routine and likes knowing what to expect. He’s not a fan of…
Alagille syndrome symptoms vary by gene mutation, study finds
People with Alagille syndrome display different disease characteristics depending on whether the disease-causing mutation is located in the JAG1 gene or the NOTCH2 gene,…
Preparing my son with Alagille syndrome, and myself, for surgery
Preparing a child for surgery is scary, as I’m being reminded. My 4-year-old son, Finley, is scheduled to have a small procedure to fix a…
Sometimes I wonder what ‘stable’ means regarding my son’s health
Rare diseases bring a lot of unknowns to the lives of patients, caregivers, and family members. We caregivers often worry about what might be looming…
New mutation props up woman’s Alagille syndrome diagnosis
An unreported mutation in the JAG1 gene was identified as the likely cause of Alagille syndrome in a 28-year-old woman, a case report from…
My son has his own understanding of his liver disease medications
Every time I meet another family raising a child with Alagille syndrome, the topic of medication comes up. I have found that parents and…
A big-boy bed has been a positive change for our son with Alagille
Bedtime has been tricky in my house over the past few months. My son Finley, who was born with Alagille syndrome, just doesn’t seem…
Scientists ID ways to aid Alagille syndrome diagnosis in infants
In infants younger than 3 months, identifying heart problems and distinctive facial features, as well as blood markers and certain liver tissue abnormalities, may aid…
Recent Posts
- New Alagille syndrome therapy named ART4 gets FDA designations
- Gene-editing approach may one day cure chronic hepatitis B: Study
- New trial data show saroglitazar works to ease liver damage in PBC
- With liver disease, getting a good night’s sleep is crucial
- New medications show promise for children with rare liver disease