Certified Global Research Member

Key Questions Answered

Global Market Outlook
In-depth analysis of global and regional trends
Analyze and identify the major players in the market, their market share, key developments, etc.
To understand the capability of the major players based on products offered, financials, and strategies.
Identify disrupting products, companies, and trends.
To identify opportunities in the market.
Analyze the key challenges in the market.
Analyze the regional penetration of players, products, and services in the market.
Comparison of major playersâ€™ financial performance.
Evaluate strategies adopted by major players.
Recommendations

Why Choose Market Research Future?

Vigorous research methodologies for specific market.
Knowledge partners across the globe
Large network of partner consultants.
Ever-increasing/ Escalating data base with quarterly monitoring of various markets
Trusted by fortune 500 companies/startups/ universities/organizations
Large database of 5000+ markets reports.
Effective and prompt pre- and post-sales support.

Pompe Disease Treatment Companies

Pompe disease is a rare genetic disorder characterized by the buildup of glycogen in cells, leading to muscle weakness and other symptoms. Companies involved in Pompe disease treatment develop and provide therapies, drugs, and supportive services to manage the condition.

Pompe Disease Treatment Key Companies Disclaimer: List of key companies in no particular orderLatest Pompe disease Companies Update

September 2023: Amicus Therapeutics has just obtained formal clearance from the U.S. Food and Drug Administration (FDA) for the combination therapy of Pombiliti (cipaglucosidase alfa-atga) and Opfolda (miglustat). The medicine is intended for the treatment of adult individuals diagnosed with late-onset Pompe disease (LOPD) who do not exhibit improvement while undergoing their current enzyme replacement therapy (ERT). Its formulation is specifically developed to enhance the absorption into muscle cells. Once inside the cellular environment, Pombiliti can undergo appropriate processing to attain its fully active and mature state, facilitating the breakdown of glycogen. Opfolda functions as an enzyme stabilizer specifically engineered to mitigate enzyme instability in the bloodstream.

October 2023: Aro Biotherapeutics, a biotechnology firm at the clinical stage, made an announcement on the enrollment of the initial participant in their Phase 1 study for ABX1100. This trial focuses on Aro's primary Centyrin-siRNA product, which aims to cure Pompe Disease, a genetic disorder. ABX1100 is an innovative compound that introduces a novel strategy for mitigating the accumulation of glycogen, which can reach hazardous levels, within the muscles of individuals diagnosed with Pompe Disease. The Phase 1 trial, now underway in Canada, will begin with a segment including normal, healthy volunteers. This segment will consist of a placebo-controlled, double-blinded study, where participants will receive increasing doses of the treatment. The purpose of this study is to test the safety, tolerability, pharmacokinetics, and several pharmacodynamic biomarkers in order to demonstrate target engagement.

List of Pompe disease Key companies in the market

Amicus Therapeutics, Inc (US)

Sanofi (France)

Audentes Therapeutics (US)

Oxyrane (UK)

Valerion Therapeutics (US)

AVROBIO, Inc. (US)

CENTOGENE AG (UK)

Pompe Disease Treatment Market Highlights:

Pompe Disease Treatment Market Size

Pompe Disease Treatment Market Trends

Pompe Disease Treatment Market Analysis

Pompe Disease Treatment Market Share

Pompe Disease Treatment Market Highlights:

Pompe Disease Treatment Market Trends

Pompe Disease Treatment Market Analysis

Pompe Disease Treatment Market Share

Pompe Disease Treatment Companies

Leading companies partner with us for data-driven Insights

Kindly complete the form below to receive a free sample of this Report

Please fill in Business Email for Quick Response

We do not share your information with anyone. However, we may send you emails based on your report interest from time to time. You may contact us at any time to opt-out.

Purchase Option
	Single User	$ 4,950
	Multiuser License	$ 5,950
	Enterprise User	$ 7,250
Compare Licenses

Tailored for You

Dedicated Research on any specifics segment or region.
Focused Research on specific players in the market.
Custom Report based only on your requirements.
Flexibility to add or subtract any chapter in the study.
Historic data from 2014 and forecasts outlook till 2040.
Flexibility of providing data/insights in formats (PDF, PPT, Excel).
Provide cross segmentation in applicable scenario/markets.