NewsUNITERARE4 days ago
The PAN Foundation's financial assistance program for phenylketonuria (PKU) — a rare genetic condition where the body can't break down a protein called phenylalanine — is currently closed. This program normally helps patients pay for treatment costs. The fund status changed on April 20, 2026.
WHY IT MATTERSIf you have PKU and were relying on PAN Foundation assistance to afford your medical care or special diet, you need to find alternative financial resources immediately since this program is no longer accepting applications.
Clinical trialCLINICALTRIALS4 days ago
Williams syndrome is a rare genetic condition that affects about 1 in every 8,000 to 12,000 people. It happens when a person is missing a small section of DNA on chromosome 7, which includes the gene that makes elastin (a protein that helps blood vessels stretch). This causes distinctive facial features and heart and blood vessel problems. Researchers are now recruiting 1,099 people with Williams syndrome or a related heart condition called supravalvular aortic stenosis to donate DNA and tissue samples to help scientists better understand these conditions.
WHY IT MATTERSThis actively recruiting biobank offers Williams syndrome and SVAS patients a direct way to contribute DNA and tissue samples to NHLBI-sponsored research that could accelerate understanding of the genetic causes and lead to new treatments.
NewsUNITERAREApr 3
The PAN Foundation's financial assistance program for phenylketonuria (PKU) — a rare genetic condition that affects how the body processes a protein called phenylalanine — is currently closed and not accepting new applications. The program previously offered up to $12,000 per year to help patients pay for treatment costs. The fund status changed on April 2, 2026.
WHY IT MATTERSIf you have PKU and were relying on this $12,000 annual assistance to afford your special diet or medications, you'll need to find alternative financial support resources immediately.
Clinical trialCLINICALTRIALSApr 1
Researchers are looking for 88 people with Fragile X Syndrome or Creatine Transporter Deficiency to test a new imaging technology that can see how the brain works. Both of these are genetic conditions that affect how the brain develops and cause intellectual disability. This study might help doctors better understand and diagnose these conditions in the future.
WHY IT MATTERSThis trial is recruiting patients with two of the most common X-linked intellectual disability disorders and may provide a new diagnostic tool that could improve how these conditions are identified and monitored.