Boston Children's Hospital

NS Pharma's parent company Nippon Shinyaku has finalized a strategic partnership with Boston Children's Hospital to develop innovative therapies for rare diseases.

Dr. Erin E. Mauney at Massachusetts General Hospital is conducting the first clinical trial examining psilocybin's effects on treatment-resistant irritable bowel syndrome (IBS).

Children with uncontrolled moderate to severe asthma experienced significantly fewer exacerbations when treated with dupilumab compared to placebo, regardless of how long they had been living with the disease.

The FDA has cleared BlackfinBio's IND application to begin a Phase 1/2 clinical trial of BFB-101, a gene therapy designed to treat Hereditary Spastic Paraplegia Type 47 (SPG47), a rare neurological disorder with no existing treatments.

Luminopia's VR-based digital therapeutic for amblyopia has received FDA clearance for children aged 8-12 years, expanding from its previous 4-7 year age indication based on robust real-world evidence.

Recent research published in JAMA predicts millions of preventable disease cases over the next 25 years if childhood vaccination rates continue to decline from their current level of under 93%.

Recent research shows that ozone pollution exposure during a child's first two years increases asthma risk by 31% at ages 4-6, highlighting the need for long-term ozone regulation.

• The FDA has approved Neurelis' VALTOCO (diazepam nasal spray) for treating seizure clusters in patients as young as 2 years old, expanding its previous indication to include younger children. • Clinical trials demonstrated VALTOCO's safety and efficacy in children aged 2-5, with 31 of 35 enrolled patients completing the 180-day safety period and no treatment-related serious adverse events reported. • The nasal spray formulation provides a significant alternative to rectal diazepam, addressing a major unmet need for caregivers managing seizures in young children.

The Lupus Research Alliance has launched its Targeted Research Program on Engineered Cell Therapies for Lupus (TRP-ECT), awarding grants to 11 researchers developing innovative cellular treatments.

The US FDA has approved BlackfinBio's investigational new drug application for BFB-101, an adeno-associated virus gene therapy targeting hereditary spastic paraplegia type 47 (SPG47).