Why I sometimes feel fear of the future for my youngest son
Parenting brings many challenges, but there’s one that’s always front and center for me: keeping my sons safe and secure. As they’re 6 and 3…
Alagille syndrome
EMA committee recommends Iqirvo approval for PBC in Europe
A European Medicines Agency (EMA) committee has recommended the approval of Ipsen‘s Iqirvo (elafibranor) as a second-line therapy for adults with primary biliary…
The latest obstacles we faced in getting my son’s blood drawn
After my wife, Dani, and I entered the world of Alagille syndrome and became advocates, we aimed to provide a view of everyday life…
Dupilumab works as itching treatment in Alagille case: Report
The use of dupilumab, an approved anti-inflammatory antibody-based treatment, worked to ease itching, known medically as pruritus, in a young woman with Alagille syndrome…
Worrying about water play for our son with Alagille syndrome
Our kids love the water. When our lovely Pacific Northwest weather finally turns warm enough, they both beg to pull the inflatable pool from storage.
A parent’s dilemma: How to get a child with Alagille to eat enough
Feeding small children is a daunting process for parents. Will they eat what’s been prepared? How much of a mess will they make? Are they…
EU initiative aims to improve early detection of rare genetic diseases
An initiative in the European Union (EU) aims to speed up the diagnosis of rare genetic disorders such as Alagille syndrome in newborns using…
Clinical genome sequencing aids in rare genetic disease diagnosis: Study
Clinical genome sequencing, or cGS, testing to detect a rare genetic disease such as Alagille syndrome, resulted in a positive diagnosis of varying disorders…
The Alagille syndrome symptoms in my son’s face don’t define him
When we had our two boys, my wife, Dani, and I would debate which of us they’d look like the most. It was a fun,…
Caregivers, don’t skimp on taking care of yourself
Parenting is hard. Add in a rare genetic disease, and the stressors change. Taking time for yourself as a caregiver is vitally important to help…
Recent Posts
- How I’m learning to cope with challenges as a rare disease parent
- IBAT inhibitors have potential to boost life quality for patients
- Trial of HBV treatment candidate now enrolling final patient group
- Cour’s CNP-104 granted FDA orphan drug status for treating PBC
- Maralixibat Phase 3 trial for cholestasis-related itch is enrolling