Maxwell and Riley Freed are twins who just celebrated their third birthday. Maxwell has a rare disease called SLC6A1. Sadly, this was a notable birthday because at 3, Maxwell will…
With 4.15 million cases across the globe, COVID-19 is at the forefront of discussion. Knowledge about the viral pandemic continues to grow each and every day. Yet there are still…
According to a story from the CureDuchenne Blog, CureDuchenne founder and CEO Debra Miller announced that the organization has recently entered into a new partnership with the biotechnology company Myosana…
by Jodee Redmond from In The Cloud Copy Rich Horgan is the founder of the Boston-based biotech non-profit, Cure Rare Diseases. The 28-year-old decided to take action over concerns about…
While genomic research presents the unique opportunity of combating diseases and gene malfunctions at the source, the practice has been incredibly controversial. Some people worry about the ethics of…
As originally discussed in Muscular Dystrophy News Today, a recent study has discovered that increasing levels of utrophin in the body can improve muscle strength, form, and function for patients…
According to a study published in the journal Neurology, a measure called the vastus lateralis fat fraction was found to be useful in predicting the age at which a patient living…
According to a story from gurufocus.com, the biotechnology company Capricor Therapeutics has recently announced the initiation of a compassionate use program for its experimental drug CAP-1002. This drug will be…
According to a story from CureDuchenne, the biotechnology company Dyne Therapeutics recently announced that CureDuchenne Ventures has made an equity investment that will support the development of all new precision…
In a Special Issue of the journal Disease Models and Mechanisms Highlights, James J. Dowling et al describes pediatric neuromuscular diseases as mostly genetic and affecting areas of the peripheral…
As previously published in Scientific American, slightly after her first birthday, Emma Larson lost the use of her legs and started experiencing difficulty crawling. She was diagnosed with spinal muscular…
Patients with congenital diseases often experience a range of uncomfortable symptoms. On one side of the world, a family with erythromelalgia, a painful condition which can cause redness and swelling…
A recent editorial focusing on NMDs (neuromuscular disorders) was published in the journal Disease Models & Mechanisms. The editorial highlights a special issue of the journal devoted to building…
Shelley Simmonds recently attended the Festival of Genomics, the largest genomics event in the United Kingdom that is quickly growing into the biggest event in the world. It involves…
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Advances in medicine have been responsible for longer overall survival in patients with neurodegenerative-neuromuscular diseases. According to a recent report in RTMagazine, an increase in the number of patients…
Janet Woodcock, Director of the FDA’s Center for Drug Evaluation and Research (CDER) recently gave an interview to the publication FDA Voices about the agency’s approval in 2019 of a…
AskBio Asklepios BioPharmaceuticals (AskBio) was first founded in 2001. It is based out of North Carolina. This company is dedicated to the research of gene therapies as potential therapeutic options…
According to a story from Charcot-Marie-Tooth News, the Muscular Dystrophy Association (MDA) may at first seem like a group that is solely focused on helping muscular dystrophy patients, but its…
According to a story from Salon, the Quality Adjusted Life Year (QALY) is a statistical measure that is used in order to calculate the value of a certain medication. First…
The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two…
PTC Therapeutics is a pharmaceutical company that has been working with Translarna, a medication intended for the treatment of Duchenne muscular dystrophy (DMD). They analyzed this treatment and presented the…
CureDuchenne has launched the CureDuchenne Biobank, a neutral, centralized and self-funded data hub that will unlock information to accelerate research. It will provide a consistent set of resources for scientific…
According to a story from BioSpace, the US Food and Drug Administration (FDA) has recently approved the drug golodirsen (marketed as Vyondys 53) as a treatment for Duchenne muscular dystrophy…
A new precision medicine center, specifically for pediatrics, has launched in Utah. This new center is the result of collaborative efforts from the Intermountain Primary Children's Hospital, Intermountain Precision Genomics,…
Researchers at the University of Pennsylvania are working on a new potential gene therapy for Duchenne muscular dystrophy (DMD). DMD is a rare form of muscular dystrophy that primarily affects…
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