A Mother’s Thoughts When Her Two-Year-Old Daughter Was Diagnosed with Acute Lymphoblastic Leukemia

Imagine the highs and lows that accompany the doctor’s announcement that your sweet, innocent two-year-old daughter has leukemia. The good news is that your daughter has the type of leukemia…

Continue Reading A Mother’s Thoughts When Her Two-Year-Old Daughter Was Diagnosed with Acute Lymphoblastic Leukemia

Patient Story: Living with Dystonia

Recently, an article highlighted one man’s life journey after being diagnosed with rare cervical dystonia. Dystonia Dystonia is a chronic movement disorder, existing in the same family as the more…

Continue Reading Patient Story: Living with Dystonia
Patient Story: Liver Transplant Allows Girl Diagnosed with Biliary Atresia to Celebrate her First Birthday
source: pixabay.com

Patient Story: Liver Transplant Allows Girl Diagnosed with Biliary Atresia to Celebrate her First Birthday

According to a recent article, a child with biliary atresia was able to celebrate her first birthday following her liver transplant. Biliary Atresia Biliary atresia is a condition occurring in…

Continue Reading Patient Story: Liver Transplant Allows Girl Diagnosed with Biliary Atresia to Celebrate her First Birthday
One Family’s Story on Being Diagnosed with Progressive Familial Intrahepatic Cholangitis
Source: Pixabay.com

One Family’s Story on Being Diagnosed with Progressive Familial Intrahepatic Cholangitis

A recent article shared the story of one family’s journey with multiple family members being diagnosed with progressive familial intrahepatic cholangitis (PFIC) after years of medical issues. Progressive Familial Intrahepatic…

Continue Reading One Family’s Story on Being Diagnosed with Progressive Familial Intrahepatic Cholangitis
Interview: Living a Full and Filling Life with Late-Onset Tay-Sachs (LOTS) Pt. 2
Stewart and Lorrie Altman

Interview: Living a Full and Filling Life with Late-Onset Tay-Sachs (LOTS) Pt. 2

In Part 1 of our interview with Lorrie and Stewart Altman, we discussed the journey to the Late-onset Tay-Sachs disease (LOTS) diagnosis, how Lorrie and Stewart's outlook shaped their lives,…

Continue Reading Interview: Living a Full and Filling Life with Late-Onset Tay-Sachs (LOTS) Pt. 2
A Discussion of von Hippel-Lindau Disease Thirty Years After Discovery
source: pixabay.com

A Discussion of von Hippel-Lindau Disease Thirty Years After Discovery

CURE® recently conducted an interview with von Hippel-Lindau (VHL) patient Joseph Heisler and his mother, Marirene. Joseph’s father was diagnosed thirty years ago with the genetic (inherited) disease. Shortly thereafter…

Continue Reading A Discussion of von Hippel-Lindau Disease Thirty Years After Discovery

Two Teenage Boys Diagnosed With ANCA-Associated Vasculitis, Part One

A group of autoimmune diseases called anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis (AAV) is the subject of a preprint under consideration at Pediatric Rheumatology. ANCA-AAV is characterized by damage to small…

Continue Reading Two Teenage Boys Diagnosed With ANCA-Associated Vasculitis, Part One
INTERVIEW: How ProQR’s Sirius and Celeste Studies Could Change the Therapeutic Landscape for USH2A-Mediated RP and Usher Syndrome (Pt. 2)
Daniel de Boer. Photo courtesy of: ProQR and FTI Consulting

INTERVIEW: How ProQR’s Sirius and Celeste Studies Could Change the Therapeutic Landscape for USH2A-Mediated RP and Usher Syndrome (Pt. 2)

If you would like to read the first part of this interview, please take a look at Part 1 here. In the first part of the interview, I spoke with…

Continue Reading INTERVIEW: How ProQR’s Sirius and Celeste Studies Could Change the Therapeutic Landscape for USH2A-Mediated RP and Usher Syndrome (Pt. 2)
A Parent’s Take on Marriage While Having a Child with Duchenne Muscular Dystrophy
source: pixabay.com

A Parent’s Take on Marriage While Having a Child with Duchenne Muscular Dystrophy

  Betty Vertin recently wrote an article discussing her experience as a parent of a child with Duchenne muscular dystrophy. She shares the lessons she has learned in order to…

Continue Reading A Parent’s Take on Marriage While Having a Child with Duchenne Muscular Dystrophy