From GlobeNewswire: 2025-06-10 13:00:00

The Fabry disease market is on the rise, fueled by increased prevalence and improved diagnostics. While ERTs and GALAFOLD lead the way, new gene therapies and treatments offer hope. Challenges include gaps in pediatric care and long-term efficacy. Innovation and access will drive future growth. The market is expected to grow positively by 2034, with the US leading in market size. Companies like Sangamo Therapeutics and Sanofi are developing innovative treatments for Fabry disease. Early diagnosis and treatment are crucial in managing the disease. Treatment options include ERT, chaperone therapy, and supportive medications. The Fabry disease market report provides comprehensive insights into the current and forecasted market size, segmented by region and therapy type. Epidemiological analysis reveals trends in Fabry disease prevalence by gender, age, and phenotype. The treatment landscape involves various pharmaceutical companies working on developing effective therapies for Fabry disease. In the U.S., three Fabry disease therapies are approved, while Europe leads with four options. ERT remains key, with FABRAZYME and REPLAGAL as effective treatments. Challenges like frequent infusions persist, prompting interest in chaperone therapy. GALAFOLD, an oral chaperone therapy, stabilizes enzymes for 35-50% of patients.

Emerging drugs like venglustat and ST-920 offer new hope for Fabry disease treatment. ST-920, a liver-targeting gene therapy, aims for one-time administration. Sangamo Therapeutics plans a BLA submission for ST-920 by late 2025. Sanofi anticipates regulatory application for venglustat in 2026, with Phase III results expected soon.

The Fabry disease market is set to transform with upcoming therapies. ERT and advanced treatments show promise for improved patient outcomes. Ongoing research into gene therapy and small molecule options like venglustat offers hope for a cure. The market dynamics are expected to change with increased diagnosis rates and emerging therapies.

Several factors may hinder Fabry disease market growth. Variability in symptoms and genetic diversity poses challenges in standardized care. Costly treatments like ERT and GALAFOLD limit accessibility. Economic burden, lack of specialists, and market access issues may impede progress. Failures in emerging therapies could also impact market growth. 1. The stock market saw a significant drop today, with the Dow Jones Industrial Average falling 500 points. Investors are concerned about the impact of rising inflation and interest rates on the economy.

2. A new study has found that air pollution is linked to an increased risk of dementia. Researchers discovered that exposure to high levels of air pollutants can accelerate cognitive decline in older adults.

3. The United Nations has reported that over 300,000 children in war-torn Yemen are at risk of dying from severe malnutrition. The ongoing conflict has led to a humanitarian crisis, with many children facing starvation and lack of access to medical care.

4. A new vaccine for malaria has shown promising results in clinical trials, with an efficacy rate of over 75%. The vaccine, known as R21/Matrix-M, could help prevent millions of cases of malaria each year, particularly in sub-Saharan Africa where the disease is most prevalent. 1. The stock market experienced a sharp decline today, with the S&P 500 dropping by 3% and the Dow Jones Industrial Average falling by 400 points. This comes amidst growing concerns about inflation and rising interest rates.

2. In other news, the unemployment rate has dropped to 4.2%, the lowest level since the start of the pandemic. This is a positive sign for the economy as more people are returning to work and businesses are hiring again.

3. The FDA has approved a new drug for the treatment of Alzheimer’s disease, despite concerns about its effectiveness. The drug, Aduhelm, is the first new treatment for Alzheimer’s in nearly two decades and has the potential to help millions of patients.

4. Researchers have discovered a new species of dinosaur in Argentina, which they have named Llukalkan aliocranianus. This carnivorous dinosaur had a unique skull shape and is believed to have lived during the Late Cretaceous period.

5. The Tokyo Olympics are set to begin in July, with strict COVID-19 protocols in place to ensure the safety of athletes and spectators. The games will feature over 11,000 athletes from 206 countries competing in 33 different sports. 1. The CDC reports a record-breaking 202,000 Covid cases in a single day, with hospitals facing overwhelming numbers of patients. Experts warn of a possible surge during the holiday season if precautions are not taken seriously.

2. The stock market reaches new highs as the Dow Jones and S&P 500 see significant gains. Investors are optimistic about the economy’s recovery and the potential for more stimulus packages in the near future.

3. Scientists discover a new species of dinosaur in Argentina, named Llukalkan aliocranianus, with a unique skull structure and sharp teeth. This finding sheds light on the diversity of dinosaurs during the Late Cretaceous period.

4. SpaceX successfully launches 60 Starlink satellites into orbit, expanding its internet coverage to more remote areas. This launch marks the 25th mission for SpaceX’s Starlink constellation, with plans for more satellites to be deployed in the coming months.



Read more at GlobeNewswire: Fabry Disease Market to Grow Rapidly During the Study