Ambitious NCATS Bespoke Gene Therapy Consortium Targets Ultrarare Diseases
The Bespoke Gene Therapy Consortium (BGTC) is an ambitious public-private partnership that aims to develop gene therapies for ultrarare diseases.
The Bespoke Gene Therapy Consortium (BGTC) is an ambitious public-private partnership that aims to develop gene therapies for ultrarare diseases.
The EveryLife Foundation for Rare Diseases pushes a legislative agenda aimed at benefitting the 30 million Americans with a rare disease.
Pediatric gastroenterologist Dr. Henry Lin describes growth, nutritional, and psychosocial considerations for clinicians managing patients with ALGS.
Dr. Henry Lin reviews common clinical presentations and management considerations for renal, skeletal, ocular, and vascular involvement in ALGS.
Pediatric gastroenterologist Henry Lin, MD, MBA, reviews how the liver and cardiac system are involved in Alagille syndrome.
Kristen Baugher knew shortly after giving birth to her daughter, Emmery, that something was wrong. Jaundiced and weak, the baby itched uncontrollably.
When it comes to rare disorders, Israel—a New Jersey-sized nation of 9.3 million that’s made headlines this year for its highly successful coronavirus vaccination campaign—is clearly an emerging powerhouse.
In August of 2003, Roberta Smith of East Tawas, Michigan, gave birth to twin daughters. It was immediately obvious that one of the girls, Cloe, was seriously ill. Jaundiced and plagued by incessant itching, the infant was diagnosed with Alagille syndrome 2 weeks later. Cloe’s disease is exceedingly rare, affecting roughly 1 in 30,000 to…
Although we are making headway in understanding and treating people living with Alagille Syndrome (ALGS) — a rare genetic disorder that manifests in a variety of ways — several questions remain. Here, Henry C. Lin, MD, pediatric gastroenterologist in the Division of Gastroenterology at Doernbecher Children’s Hospital, Oregon Health & Science University, shares his perspective…
Under COVID-19’s lingering shadow, the National Organization of Rare Disorders (NORD) has presented its 2021 Rare Impact Awards to 24 pharmaceutical firms, researchers, politicians, and nonprofit groups for their efforts over the past year on behalf of patients with such illnesses.