Blu Genes supports gene therapy at University of Wisconsin-Madison. Gene therapy protects against motor neuron disease.

Anjon Audhya The mutation is in the Trk-fused gene, which typically facilitates the transportation of...

Taylor Swift tickets being auctioned off to help fund for Ottawa boy with rare disease

A pair of Taylor Swift tickets are being auctioned off in Ottawa to help fund...

Saskatchewan artist fundraising to help family whose son has genetic disease

A Regina based artist is using his platform to fundraise in hope of helping a...

Family with child affected by SPG4 visits UMass Chan researchers developing gene therapy

A family from Ottawa, Canada, seeking a cure for spastic paraplegia type 4 (SPG4), a...

Ottawa community helping fundraise for boy with rare disease

Ottawa came together in Stittsville on Saturday, raising more than $15,000 to help fund a...

FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy

Today, the U.S. Food and Drug Administration approved Lenmeldy (atidarsagene autotemcel), the first FDA-approved gene...

Gene Therapy Allows an 11-Year-Old Boy to Hear for the First Time

The genetic treatment targeted a particular kind of congenital deafness and will soon be tried...

Genetic test offers hope for undiagnosed kids, but few insurers offer to pay. Florida Medicaid will

Just 48 hours after her birth in a Seattle-area hospital in 2021, Layla Babayev was...

The science behind screening for Tay-Sachs disease

Shari Ungerleider lost her son to Tay-Sachs disease and spent the last 27 years working...

Canadian family raised $3.5M to develop individualized gene therapy for son’s rare condition

The family is cautiously optimistic about the improvements they are seeing in Michael, 4, since...