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...

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...

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...

Lysosomes and their role in Alzheimer’s disease, Duchenne Muscular Dystrophy, Tay-Sachs Disease

U–M researchers reveal how a mutated protein acts as risk factor in Parkinson’s disease Reviewed...

Parents spark breakthrough gene therapy for devastating child disease ignored by big pharma

Exclusive: For more than a century there has been no cure, no treatment and no...

First gene therapy for Tay-Sachs disease successfully given to two children

Two babies have received the first-ever gene therapy for Tay-Sachs disease after over 14 years...

Medical Journal Publication – AAV gene therapy for Tay-Sachs Disease.

Tay-Sachs disease (TSD) is an inherited neurological disorder caused by deficiency of hexosaminidase A (HexA)....

UMass Chan launches Translational Institute for Molecular Therapeutics

Study supported by Blu Genes Published in Nature Medicine Blu Genes Foundation made a commitment...