- Home
- Primary Care
- Transforming Gaucher Disease Care: Venglustat Clinical Data Highlights

15h3 min read
Medical Article
Gaucher disease (GD), a rare genetic disorder inherited via the autosomal recessive mode, is the most prevalent lysosomal storage disorder (1). Mutations in the glucocerebrosidase (GBA) gene lead to GD manifestations (1). Nearly 300 GBA mutations have been identified, including missense, nonsense, splice junction, deletion, insertion, gene fusion,

Transforming Gaucher Disease Care: Venglustat Clinical Data Highlights
18 Reached
Similar Content

ACC, AHA and ESC Recommendations for Heart Failure Management
2323 Reached29 Comments2 Likes

Catheter Ablation for Atrial Fibrillation with HF
691 Reached

Single-incision Laparoscopy for Gastrostomy
407 Reached2 Comments1 Likes

Pancytopenia, a rare presentation of hyperthyroidism
826 Reached2 Comments

Intestinal Obstruction and Ileocolic Fistula due to Gossypiboma
861 Reached30 Comments15 Likes