Cincinnati Children's Hospital Medical Center

Amicus Therapeutics to Present Extensive Research on Fabry and Pompe Diseases at WORLDSymposium 2025

4 months ago

• Amicus Therapeutics will showcase 16 presentations at WORLDSymposium 2025, featuring comprehensive research on migalastat for Fabry disease and cipaglucosidase alfa plus miglustat for Pompe disease. • Key studies include long-term patient outcomes from the FollowME Fabry Pathfinders registry and real-world evidence of treatment effectiveness in both rare diseases. • Notable research highlights treatment satisfaction, quality of life improvements, and clinical outcomes in patients switching from existing therapies to Amicus' novel treatment approaches.

Enzyvant's RVT-802 Receives Landmark Dual Fast-Track Status from FDA for Complete DiGeorge Syndrome

4 months ago

• Enzyvant has achieved a historic milestone as the first company to receive simultaneous Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA for RVT-802. • RVT-802, a tissue-based therapy for complete DiGeorge Syndrome, has shown promising preliminary results with over 70% survival rate in treated infants. • The Swiss biotech startup plans to file a Biologics License Application (BLA) in the first half of 2018, marking a significant advancement in rare disease treatment.

FDA Expands Impella Heart Pump Indication to Include Pediatric Patients

5 months ago

• The FDA has granted premarket approval to Johnson & Johnson MedTech's Impella 5.5 and CP heart pumps for pediatric patients with acute decompensated heart failure and cardiogenic shock. • The Impella CP is approved for children weighing at least 52kg, while Impella 5.5 is approved for those weighing at least 30kg, offering temporary ventricular support. • These left-sided heart pumps unload the heart's left ventricle, allowing it to rest while maintaining oxygenated blood flow throughout the body, facilitating heart recovery. • Johnson & Johnson MedTech collaborated with the ACTION network to gather real-world data supporting the use of Impella pumps in pediatric patients.

NIH Launches Influenza Human Challenge Study to Advance Vaccine Development

6 years ago

• The National Institute of Allergy and Infectious Diseases (NIAID) has initiated a clinical trial where healthy adults will be deliberately infected with influenza virus under controlled conditions at four research sites across the US. • The study aims to assess how pre-existing influenza antibodies affect the timing, magnitude, and duration of flu symptoms, with up to 80 volunteers aged 18-50 participating in the trial. • Participants will receive a nasal spray containing the InfluenzaA/Bethesda/MM2/H1N1 strain and remain under observation for at least seven days, with preliminary results expected by May 2020.