Seven previously unreported mutations linked to type 1 Gaucher disease were identified in a study analyzing the genetic code…
Articles by Marisa Wexler MS
One-time treatment with the gene therapy FLT201 (avigbagene parvec) may help control Gaucher disease without the need for…
People with Gaucher disease are not at a higher risk of developing cancer, but their risk of blood cancers…
In Gaucher disease, the toxic buildup of a fatty molecule called glucosylsphingosine (lyso-Gb1) leads to issues with how nerve…
Sharp Therapeutics is gearing up to ask the U.S. Food and Drug Administration (FDA) for permission to begin testing…
Casma Therapeutics has selected its first experimental therapy candidate, dubbed CSM-101, which will be developed to treat Gaucher’s…
FLT201, a gene therapy for Gaucher disease type 1 currently in clinical testing, led to sustained increases in…
Treatment with the experimental gene therapy FLT201 led to sustained clinical benefits that lasted up to 21 months for…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to two gene therapies that M6P Therapeutics…
Cerdelga (eliglustat) safely and effectively treated three adults with Gaucher disease type 1 (GD1), who switched from long-term use…