Myopathies News and Research

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Study reveals an alternative mechanism for muscle tissue repair after physiological damage

Muscle is known to regenerate through a complex process that involves several steps and relies on stem cells.

21 Oct 2021

Study unravels new mechanism for muscle repair after physiological damage

Muscle is known to regenerate through a complex process that involves several steps and relies on stem cells. Now, a new study led by researchers at Pompeu Fabra University (UPF, Spain)/Centro Nacional de Investigationes Cardiovasculares (CNIC, Spain)/CIBERNED (Spain) and Instituto de Medicina Molecular João Lobo Antunes (iMM, Portugal), published on 15 October in the journal Science, describes a new mechanism for muscle repair after physiological damage relying on the rearrangement of muscle fiber nuclei, and independently of muscle stem cells.

15 Oct 2021

FDA grants seven years of marketing exclusivity for Octapharma’s Octagam 10%

Octapharma USA today announced the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD) has awarded seven years of marketing exclusivity for Octagam® 10% [Immune Globulin Intravenous (Human)], the first and only intravenous immunoglobulin (IVIg) to be indicated for the treatment of adult dermatomyositis (DM).

31 Aug 2021

New initiative to reduce treatment delays for patients with rare diseases

The Treatabolome project is a research initiative to develop a freely available, interoperable online platform dedicated to disseminating rare disease and gene-specific treatment information to healthcare professionals regardless of their level of specialized expertise.

1 Jun 2021

Researchers identify mechanism by which dermatomyositis may develop in humans

Dermatomyositis is an idiopathic inflammatory myopathy that has been regarded as an autoimmunity-based disorder, although its pathogenesis remains unclear.

8 Apr 2021

COVID-19 lockdown restrictions reduced light activity related to socializing and work

A study which looked at activity levels before and during the COVID-19 pandemic has found lockdown restrictions significantly reduced light activity associated with socializing and work.

22 Mar 2021

Study offers renewed hope for children suffering from myofibrillar myopathies

Stephen Greenspan and Laura Zah were devastated when they learned their son Alexander had a rare genetic mutation, which causes a deadly neuromuscular disease with no known treatment or cure.

14 Oct 2020

Study uncovers a promising new therapeutic approach for Duchenne muscular dystrophy

Scientists at Sanford Burnham Prebys Medical Discovery Institute, Fondazione Santa Lucia IRCCS, and Università Cattolica del Sacro Cuore in Rome have shown that pharmacological (drug) correction of the content of extracellular vesicles released within dystrophic muscles can restore their ability to regenerate muscle and prevent muscle scarring (fibrosis).

15 Sep 2020

Scientific evidence shows GLDH is a safe biomarker for drug-induced liver injury

Critical Path Institute (C-Path) announced today that the Biomarker Qualification Program (BQP) at the Center for Drug Evaluation and Research (CDER) from the U.S. Food and Drug Administration (FDA) issued a positive response to the Qualification Plan (QP) for glutamate dehydrogenase (GLDH) as a safety biomarker for drug-induced liver injury (DILI), developed by C-Path's Predictive Safety Testing Consortium (PSTC) and Duchenne Regulatory Science Consortium (D-RSC).

22 Jul 2020

Combination of exercise and cell-aging drug could help people with chronic inflammatory myopathy

Exercise can only improve strength in muscle-degenerating diseases when a specific type of muscle cell ages, report a Hokkaido University researcher and colleagues with Sapporo Medical University in Japan. Their findings utilizing mice models were published in the journal Nature Communications.

31 Mar 2020

Study demonstrates the concept of systemic gene therapy treatment for gamma-sarcoglycanopathy

Gamma-sarcoglycanopathy is one of the most common limb-girdle myopathies.

25 May 2019

Researchers discover two proteins essential for development of skeletal muscle

All vertebrates need muscles to function; they are the most abundant tissue in the human body and are integral to movement.

12 Nov 2018

MDA announces 34 new grants totaling $9.9 million to accelerate neuromuscular research

The Muscular Dystrophy Association today announced the award of 34 new grants totaling more than $9.9 million for its summer round of funding. These new grants represent a continued commitment by MDA to fund groundbreaking research that will accelerate treatments and cures for the more than 40 diseases in its program.

24 Aug 2018

Study provides vital information to develop successful PI5P4K inhibitors for p53-mutant cancers

One major hallmark of cancer cells is their ability to adapt to stressful conditions such as nutrient deprivation. Rapidly growing tumor cells must compete for the ever-diminishing supply of nutrients in the surrounding environment to survive and proliferate. Targeting these adaptive mechanisms represents a promising approach for cancer therapeutics.

3 May 2018

Myopathies News and Research

Study reveals an alternative mechanism for muscle tissue repair after physiological damage

Study unravels new mechanism for muscle repair after physiological damage

FDA grants seven years of marketing exclusivity for Octapharma’s Octagam 10%

New initiative to reduce treatment delays for patients with rare diseases

Researchers identify mechanism by which dermatomyositis may develop in humans

COVID-19 lockdown restrictions reduced light activity related to socializing and work

Study offers renewed hope for children suffering from myofibrillar myopathies

Study uncovers a promising new therapeutic approach for Duchenne muscular dystrophy

Scientific evidence shows GLDH is a safe biomarker for drug-induced liver injury

Combination of exercise and cell-aging drug could help people with chronic inflammatory myopathy

Study demonstrates the concept of systemic gene therapy treatment for gamma-sarcoglycanopathy

Researchers discover two proteins essential for development of skeletal muscle

MDA announces 34 new grants totaling $9.9 million to accelerate neuromuscular research

Study provides vital information to develop successful PI5P4K inhibitors for p53-mutant cancers

Removal of single mitochondrial protein leads to severe inflammation

New, sensitive screening test may allow clinicians to accurately detect Pompe disease

New research offers crucial step towards understanding cause of myopathy

Researchers discover small protein essential for formation of skeletal muscle

Some WADA-banned substances may have potential medical applications

Key gene controls ability of adult stem cells to regenerate muscle after injury, study finds

How ELRIG Is Helping Mid-Career Scientists Navigate Leadership, Career Change and Redundancy

The role of UV-Vis in biopharma QC

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