Pompe Disease - Latest News and Research Updates

Health News Posted on April 11, 2021 -

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Medindia provides you with the latest news and research breakthroughs on Pompe Disease. Please find 74 such items on this topic.

Enzyme Replacement Therapy Launched by Takeda in India

Global biopharmaceutical company Takeda announced the launch of the enzyme replacement therapy for ’lysosomal storage disorders’ (LSDs), in an attempt to expand its portfolio of rare disease therapies in India. "Under ’lysosomal storage ...

Overweight Teens More Likely to Develop Heart Muscle Disease in Adulthood

Being overweight in adolescence may increase the risk of developing heart muscle disease (cardiomyopathy) later during adulthood, reports a new study. The risk of developing cardiomyopathy, which often leads to heart failure, increased in adult ...

Manuka Honey Kills Drug-resistant Bacteria in Genetic Disease

Manuka honey could act as an alternative to antibiotic to treat drug-resistant respiratory infections in a genetic disorder called Cystic Fibrosis (CF), according to Dr. Rowena Jenkins and Dr. Aled Roberts of Swansea University. Using lung ...

Heavy Teens may Face Higher Heart Muscle Disease Risk as Adults

Being obese and overweight in adolescence may increase the risk of developing heart muscle disease later during adulthood, reports a new study. A large study of Swedish men found that those who were even mildly overweight around age 18 were ...

AI can Identify Risk of Genetic Disease Causing High Cholesterol

Highlights: New AI-based technology can detect patients with familial hypercholesterolemia – a genetic disease that causes high blood cholesterol The technology can accurately differentiate between different categories of patients with high ...

Phenylketonuria, the Genetic Disease Healed Using Genome Editing

CRISPR/Cas9 system was found to show promising results to correct both mutated genes in the liver cells and thus heal the metabolic disorder phenylketonuria. Parents of newborns may be familiar with the metabolic disorder phenylketonuria: in ...

New Gene Therapy Medication can Treat Muscle Disease in Children

Gene therapy medication, Spinraza can treat the most severe form of neuromuscular disease, spinal muscular atrophy (SMA) that is mostly seen in children from 6 to 18 months of age, finds a new study. The findings of the study are published in the ...

Scientists Identify Cause of AEC Syndrome, a Severe Genetic Disease

Goethe University Frankfurt scientists in collaboration with a research group from the University of Naples Federico II have now identified that AEC syndrome resembles diseases such as Alzheimer's, Parkinson's or ALS more closely than it does other ...

Genetic Mutation Linked To Muscle Disease - Myopathy

Myopathies are diseases that prevent muscle fibres from functioning properly, causing muscular weakness. At present, there is no single treatment for the disease, as it can develop via a number of different pathways. Pioneering research using ...

Many Patients Carry Rare Genetic Disease Risks

Whole genome sequencing reveals the complete DNA make-up of an organism, enabling us to better understand variations both within and between species. This in turn allows us to differentiate between organisms with a precision that other ...

Mechanism Behind Rare Genetic Disease in Babies Revealed

A rare but severe genetic disease that affects babies, children, and adults is caused by gene mutations that destroy the function of a protein but the physiological function was little understood. In Hyaline Fibromatosis Syndrome (HFS) a glassy ...

Facial Recognition Software Helps to Diagnose Rare Genetic Disease

Facial recognition software has been successfully used to diagnose a rare, genetic disease in Africans, Asians and Latin Americans, finds a research team at the National Human Genome Research Institute (NHGRI), part of the National Institutes of ...

New Insight into Odor Producing Rare Genetic Disease

Highlights Mutations in a single gene, FM03, has been atrributed to the cause of trimethylaminuria (TMAU), which is characterized by fish-like odor symptoms. But some people who report fish-like odor symptoms, do not have severely ...

Alpha-lipoic Acid Prevents Kidney Stones in Rare Genetic Disease

Highlights Alpha-lipoic acid (ALA) is an anti-oxidant which naturally occurs in the body and in spinach, rice bran, brussel sprouts, organ meat. ALA is also used as a supplement to treat nerve-related symptoms of diabetes and prevents ...

Rare Childhood Genetic Disease Linked With a Major Cancer Gene

Fanconi anemia, a rare childhood genetic disease, is characterized by birth defects, bone marrow failure and increased cancer risk. About one in 150,000 children in the United States is born with Fanconi anemia. An important molecular link ...

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Extraordinary Measures to Tackle Pompe Disease Continue to be Taken by UF Researchers

University of Florida researchers are hopeful that gene therapy will help patients in the late stages of Pompe disease breathe on their own.

Deadly Muscle Disease may Originate in the Nervous System

A severe form of muscular dystrophy in children known as Pompe disease could originate in the brain and not muscles

Alglucosidase

Alglucosidase is an enzyme replacement therapy used for treating Pompe disease, a rare metabolic disorder that damages the muscles and nerves caused due to the accumulation of glycogen in the lysosome. This medication is a glycoprotein, ...