February, the rare shortest month of the year, also marks the month for rare liver diseases. Through webinars, conferences, research, and educational materials, the rare liver disease community joins for this year’s Rare Aware campaign to advocate for better health outcomes for our patients. There’s plenty happening amongst the strong network of organizations supporting patients with rare liver diseases.
Autoimmune Hepatitis Association Held Externally-Led Patient Focused Drug Development Meeting (EL-PFDD) on AIH
On January 27, the Autoimmune Hepatitis Association led an externally-led patient focused drug development meeting (EL-PFDD) on Autoimmune Hepatitis, focusing to provide the US FDA, drug developers, doctors, and academic researchers opportunities to hear from AIH patients about the impact the disease has on their health and daily life. During this time, patients were able to share their opinions and goals for treatment in order to demonstrate the need for new therapies that work for all patients and have fewer side effects. View the recording here.
The Orphan Drug Act (ODA), 40 Years Later
In January, the rare disease community celebrated the 40th anniversary of the Orphan Drug Act (ODA), a significant law that encourages the creation of treatments and cures for rare diseases. A dedicated group of patient advocates and forward-thinking legislators from both parties worked tirelessly to pass this legislation, which was signed into law by Ronald Reagan 40 years ago. It enables pharmaceutical companies to more easily develop therapies for rare diseases, which were previously considered unprofitable. As a result of the ODA, around 700 new drugs and biologics have been developed and approved, offering life-saving and life-changing benefits to thousands of people affected by rare diseases.
BARE Inc. to Launch New Biliary Atresia (BA) Patient Registry in 2023
This registry will allow families from all around the US, regardless of geography, to participate in the improvement of BA treatments and research. Through the registry, being deployed in collaboration with Sanford Health-CORDS, centers and liver teams of all sizes will have access to this network to hopefully bridge the gap for future research for BA patients.
New Guidelines on Pediatric Obesity Reflect Growing Concerns about Its Long-Term Health Effects
The American Academy of Pediatrics has updated its clinical practice guidelines for evaluating and treating children and teens with obesity. The new guidelines take into account the complex factors that contribute to childhood obesity, including genetic, physiologic, socioeconomic, and environmental factors. They recommend that doctors screen regularly for obesity using body mass index (BMI) as a measure and that they take a holistic approach to treatment, including counseling on nutrition and physical activity and addressing underlying issues such as access to healthy foods and safe places to play. The goal is to help children achieve and maintain a healthy weight and reduce the risk of obesity-related health complications in the future.
Upcoming GLI Events as part of #RareAware:
Register to attend the remaining Roundtables
- February 13 – Parents and Partners
- February 20 – Patient Advocacy in Rare Liver Diseases
- February 15 – Parenthood in Rare Liver Diseases
- February 22 – Maternal and Neonatal Care in Italy and Cameroon
View past episodes and other recordings on GLI’s YouTube channel.
For more information about the Pediatric and Rare Liver Diseases Council or to learn more about joining, please visit https://globalliver.org/pediatric-rare-liver-diseases-council/ or email email@example.com.