The first COPD launches are underway. Pharma launches increased 43.8% to 847 million euros, led by Altuviiio (hemophilia drug), Nexviazyme (Pompe disease), Sarclisa (multiple myeloma), and ...

It offers oral therapy for adults with Fabry disease, and a unique two-part treatment designed for adults with late-onset Pompe disease. FOLD ranks 3rd on our list of the best Guru stocks.

AB-1002 is one of several clinical-stage gene therapy programmes being run by AskBio, along with candidates for Pompe disease, Parkinson’s disease, Huntington’s disease, multiple system ...

I served on this committee from 2011 to 2017. During this time, the committee added Pompe Disease, mucopolysaccharidosis type 1 and X-linked adrenoleukodystrophy to the recommended uniform ...

Bladder cancer is one of the more common forms of cancer. According to the Centers for Disease Control and Prevention, about 57,000 men and 8,000 women are diagnosed with bladder cancer annually ...

Taiwanese patients with autoimmune disease-associated neuromyelitis optica spectrum disorder (AD-NMOSD) showed significant differences compared to patients with primary NMOSD (p-NMOSD), according to a ...

Macie, now 5, has so far shown no symptoms of Pompe disease. Caitlin Harris’s pregnancy with her second child, Macie, was uneventful. A prenatal genetic screening at her OB/GYN’s office revealed no ...

Attendees at a roundtable on gene therapy in rare disease at the World Orphan Drug Congress USA 2025 (Photo by Riya Ajmera) The most extensively discussed topic during the roundtable was the need for ...

DYNE-251 was also granted U.S. Food and Drug Administration (FDA) orphan drug and rare pediatric disease designations in March 2023. Key Milestones for the DELIVER Trial Dyne continues to pursue ...

While autophagy is relevant to many biological processes and often serves as a gatekeeper of homeostasis, when something goes wrong it can either lead to disease or be activated to fight against it.

Sjögren’s is a chronic, autoimmune disease that causes dryness of the eyes, mouth and other body parts. In an autoimmune disease, the immune system erroneously attacks healthy tissue, causing ...