Breaking WORLD PHARMA NEWS

FDA approves Roche's Evrysdi tablet as first and only tablet for Spinal Muscular Atrophy (SMA)

Roche (SIX: RO, ROG; OTCQX: RHHBY), announced that the U.S. Food and Drug Administration (FDA) has approved a New Drug Application (NDA) for an Evrysdi® (risdiplam) tablet for people living with spinal muscular atrophy (SMA). Evrysdi is the only non-invasive disease-modifying treatment for SMA. The 5 mg Evrysdi tablet can either be swallowed whole or dispersed in water.

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Daily drug captures health benefits of high-altitude, low-oxygen living

For the average person, living at high altitude - where oxygen is sparser than at sea level - can have health benefits ranging from lower rates of heart disease to increased endurance. But for those born with inherited mitochondrial diseases, who rarely survive past childhood, low-oxygen air like that found at mountain peaks could be life-saving, potentially extending their lifespan and eliminating their symptoms.

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AskBio Receives FDA Regenerative Medicine Advanced Therapy designation for Parkinson's disease investigational gene therapy

AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that investigational gene therapy AB-1005 for the treatment of Parkinson's disease (PD) has been granted Regenerative Medicine Advanced Therapy (RMAT) designation from the United States Food and Drug Administration (FDA).

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Common drug shows promise against rare type of heart disease

A research team co-led by scientists at the University of Arizona College of Medicine - Tucson found that an osteoporosis drug might counter a rare genetic mutation underlying a type of heart disease. The results were published today in the Journal of Clinical Investigation and could have implications for treating other rare diseases.

Dilated cardiomyopathy is a group of disorders defined by weak cardiac muscles.

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NUS Medicine pioneers ground-breaking technique to deliver drugs directly to the brain

Scientists at the Yong Loo Lin School of Medicine, National University of Singapore (NUS Medicine) have developed a ground-breaking technique to deliver therapeutic molecules directly to the brain, bypassing the blood-brain barrier.

Led by Dr Haosheng Shen, lead researcher from the Synthetic Biology Translational Research Programme, NUS Medicine and the NUS Synthetic Biology for Clinical and Technological Innovation (SynCTI) this novel approach utilises a naturally occurring nasal bacterium, Lactobacillus plantarum (Lp),

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An enzyme to disarm tumors

When a tumour develops, it creates a structure around itself called the tumour stroma, within which blood and lymphatic vessels ensure nutritional and respiratory biological exchanges. Lymphangiogenesis, i.e. the development of lymphatic vessels, is generally associated with a poor prognosis, as it favours the spread of metastases to other organs. By studying the cells that make up the wall of lymphatic vessels, a team from the University of Geneva (UNIGE) has made an unexpected discovery: an enzyme they express appears to play a key role in supporting immune cells, particularly when they are activated by anti-tumour treatments.

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PanK4 identified as a regulator of glucose and lipid metabolism

Physical activity is not only important for fitness, but also for health. This is confirmed by a new study conducted under the direction of Prof. Dr. Maximilian Kleinert at the German Institute of Human Nutrition Potsdam-Rehbrücke (DIfE). His team discovered that the protein PanK4 plays a crucial role in the energy metabolism of skeletal muscles. It regulates glucose uptake and fatty acid oxidation and is activated by physical exercise. The study suggests that PanK4 could be a promising approach for the treatment of metabolic diseases such as type 2 diabetes.

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Why antibiotics can fail even against non-resistant bacteria

Antibiotics are indispensable for treating bacterial infections. But why are they sometimes ineffective, even when the bacteria are not resistant? In their latest study published in the journal Nature, researchers from the University of Basel challenge the conventional view that a small subset of particularly resilient bacteria are responsible for the failure of antibiotic therapies.

In certain infectious diseases caused by bacteria, antibiotics are less effective than expected. One example is infections caused by Salmonella bacteria, which can lead to illnesses such as typhoid fever.

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New treatment offers quick cure for common cause of high blood pressure

Doctors at Queen Mary University of London, Barts Health NHS Trust, and University College London have led the development of a simple, minimally invasive Targeted Thermal Therapy (Triple T) that has the potential to transform medical management of a common, but commonly overlooked, cause of high blood pressure.

This breakthrough, published today in The Lancet, could, after further testing, help millions of people worldwide who currently go undiagnosed and untreated.

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Common antibiotic could treat inflammatory bowel disease

An antibiotic used to treat infective diarrhoea could be an effective drug for a type of inflammatory bowel disease, a new study has found.

Results published in the Journal of Crohn's and Colitis revealed that an antibiotic called vancomycin may also be effective in treating people who have a specific type of inflammatory bowel disease (IBD), which develops in the context of an incurable autoimmune liver disease called primary sclerosing cholangitis (PSC).

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