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DelveInsight Estimates a Promising Picture of Pompe Disease

The Pompe Disease Pipeline is evolving with novel mechanisms of action (MoAs) that will bring positive changes in the disease pipeline landscape.. ....

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Title: Global Ice Therapy Machines Market Expected to Reach USD 367.8 Million by 2027, With a CAGR Of 3.2%

Title: Global Ice Therapy Machines Market Expected to Reach USD 367.8 Million by 2027, With a CAGR Of 3.2%
prnewswire.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from prnewswire.com Daily Mail and Mail on Sunday newspapers.

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Newborn screening for rare diseases caught up in red tape

Newborn screening for rare diseases caught up in red tape
Newborn screening for rare diseases caught up in red tape
The call to test newborns for rare, but treatable diseases is growing. A FOX6 investigation finds the bureaucratic marathon proposed rules must endure could result in deadly delays.
MILLADORE, Wis. - The call to test newborns for rare but treatable diseases is growing. However, the process for approving those diseases is long and cumbersome. A FOX6 investigation finds the bureaucratic marathon proposed rules must endure could result in deadly delays.
By the time Gwendolyn Clubb was born in August 2017, her parents already had five other seemingly healthy children, so the big family from the tiny Wisconsin village of Milladore was not prepared for the call they received about a week after she was born. ....

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Amicus Therapeutics Announces Upcoming Presentations at the 16th International Congress on Neuromuscular Diseases

Amicus Therapeutics Announces Upcoming Presentations at the 16th International Congress on Neuromuscular Diseases
biospace.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from biospace.com Daily Mail and Mail on Sunday newspapers.

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Global Pompe Disease Pipeline Insight Report 2020: Disease Pipeline Analysis, Therapeutic Assessment, Unmet Needs, & Impact of Drugs – ResearchAndMarkets.com

ResearchAndMarkets.com’s offering.
The “Pompe Disease – Pipeline Insight, 2020,” report provides comprehensive insights about 30+ companies and 30+ pipeline drugs in the Pompe Disease pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the rapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Treatment
Enzyme replacement therapy (ERT) is an approved treatment for all patients with Pompe disease. It involves the intravenous administration of recombinant human acid alpha-glucosidase (rhGAA). This treatment is called Lumizyme (marketed as Myozyme outside the United States), and was first approved by the U.S. Food and Drug Administration (FDA) in 2006. ....

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