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In total, 10 have been published by Pompe Disease News which Ground News has aggregated in the past 3 months.

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Top Pompe Disease News News

Nurses’ pay severely eroded since 2010 – study

NHS · United Kingdom

Nurses’ pay severely eroded since 2010 – study

67% Left coverage: 9 sources

Welcome to the 2nd Raw Materials for Cell & Gene Therapy Summit!

Welcome to the 2nd Raw Materials for Cell & Gene Therapy Summit!

100% Center coverage: 1 sources

Welcome to the 2nd Raw Materials for Cell & Gene Therapy Summit! Sara.Scarpinati Wed, 09/07/2025 - 15:45 Bookmark this See the Story

Breathing problems, muscle weakness lead to LOPD diagnosis

Mississippi · Mississippi

Breathing problems, muscle weakness lead to LOPD diagnosis

A 43-year-old woman who was experiencing breathing problems and muscle weakness was eventually diagnosed with late-onset Pompe disease (LOPD) through genetic testing, highlighting the challenges of diagnosing the condition due to its rarity and nonspecific symptoms, according to a study. “It is important to consider late-onset Pompe disease in adults presenting with unexplained respiratory insufficiency and progressive muscle weakness,” clinicia…Read Article

Blindspots

Nurses’ pay severely eroded since 2010 – study

No coverage from Right Sources

Nurses’ pay severely eroded since 2010 – study

Amicus Therapeutics Announces Approval of Pombiliti® (cipaglucosidase alfa) + Opfolda® ...

100% Center coverage: 1 sources

PRINCETON, N.J., and TOKYO, June 25, 2025 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced that Japan's Ministry of Health, Labour and Welfare (MHLW) has approved Pombiliti (cipaglucosidase alfa) + Opfolda (miglustat) for the treatment of adult patients with…Japan · Japan See the Story

Screening people with muscle weakness, damage identifies LOPD

Screening people with unexplained muscle weakness or elevated levels of muscle damage markers can help to identify people with late-onset Pompe disease (LOPD), a study found. The study, “Screening for Pompe disease in Serbian patients with limb-girdle muscle weakness,” was published in Clinical Neurology and Neurosurgery. LOPD is characterized by Pompe disease symptoms that develop after the first year of life. An earlier age at symptom onset is…Serbia · Serbia Read Article

Pompe disease rates vary by region; ethnicity seen playing role

Estimates of the number people diagnosed with Pompe disease vary widely across regions, with notably higher rates in populations of African descent, a study review found. The researchers attributed the variability to underlying genetic differences as well as inconsistencies in diagnostic criteria, newborn screening methods, and disease classification depending on geographic region, factors that make it difficult to estimate Pompe disease rates w…Read Article

Substrate-reducing ASOs show promise in Pompe mouse model

A new type of substrate reduction therapy composed of antisense oligonucleotides, or ASOs, effectively reduces glycogen accumulation in the skeletal muscles, or those attached to bones, of a Pompe disease mouse model, a study found. When combined with standard enzyme replacement therapy (ERT), which showed limited efficacy in skeletal muscles, motor performance improved further than with the ASO alone. “These findings highlight the potential of …Irvine, California · Irvine Read Article

Gene, enzyme replacement improve mice's breathing, limb strength

Gene and enzyme replacement therapies for Pompe disease improved breathing and limb strength in a mouse model of the disease, a study showed. The researchers said the improvement was associated with glycogen clearance in the tongue, the lungs, and the diaphragm, the main muscle involved in respiratory control. Glycogen is a stored form of glucose, or sugar — the body’s main source of energy — that accumulates to toxic levels in Pompe patients. B…Durham, North Carolina · Durham Read Article

Short-distance walking speed test less demanding option in Pompe...

A test that measures walking speed over 10 meters (33 feet) offers a simpler and less demanding alternative to the commonly used six-minute walk test for evaluating walking performance in Pompe disease, and may be a better option for patients who have difficulty covering longer distances. In a French study with adults and adolescents being treated with Sanofi‘s Myozyme (alglucosidase alfa) — marketed as Lumizyme in the U.S. — researchers found t…Mississippi · Mississippi Read Article

Study: Nervous system involvement is common in Pompe disease

Involvement in the brain and spinal cord’s white matter is common in Pompe disease, particularly in the infantile-onset form, according to a review of published studies. Other affected parts of the brain and spinal cord, or central nervous system (CNS), were the gray matter and blood vessels in the brain, with symptoms including hearing impairment, seizures, strokes, hemorrhages, and cognitive delays. “Standardized protocols are needed to charac…Italy · Italy Read Article

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