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Launch Imminent: SpaceX Mission to the Moon Will Have Embry-Riddle Payload Onboard – ERAU News

Posted: Published on February 20th, 2024

*Update (Feb. 14): After this story's original publication, the SpaceX launch was rescheduled to 1:05 a.m. Thursday, Feb. Continue reading →

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AGAMREE: A Promising Treatment for Duchenne Muscular Dystrophy – Medriva

Posted: Published on February 20th, 2024

Duchenne muscular dystrophy (DMD) is a debilitating condition that predominantly affects males, causing progressive muscle degeneration and weakness. Continue reading →

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Muscular Dystrophy: Symptoms, Types, Causes, and Treatment – Metropolis Healthcare

Posted: Published on February 20th, 2024

What is Muscular Dystrophy? Muscular dystrophy comprises a group of conditions causing progressive muscle weakness and reduced flexibility Continue reading →

Posted in Muscular Dystrophy Treatment | Comments Off on Muscular Dystrophy: Symptoms, Types, Causes, and Treatment – Metropolis Healthcare

Opinion | Gene Therapies Could Transform Rare Diseases. Are We Holding Them Back? – The New York Times

Posted: Published on February 20th, 2024

On Jan. 8, 2020, as I was parking my car, I got a long-awaited phone call from one of my sons doctors Continue reading →

Posted in Muscular Dystrophy Treatment | Comments Off on Opinion | Gene Therapies Could Transform Rare Diseases. Are We Holding Them Back? – The New York Times

Reassessing the Process of Drug Development for Duchenne Muscular Dystrophy: Dongzhe Hong, PhD – Neurology Live

Posted: Published on February 20th, 2024

WATCH TIME: 3 minutes "One possibility is that prescribing physicians arent aware of the age range that received the drug in its pivotal trials or have knowledge on the FDA-approval process. When patients receive the drug at a later stage of their DMD, it might impact the predicted effectiveness of the medications." For years, Duchenne muscular dystrophy (DMD) was mainly treated with a combination of corticosteroids, assistive devices, and cardiac and respiratory support until 2016, when the exon-skipping therapies began to emerge. The first approved therapy was eteplirsen (Exondys 51; Sarepta Therapeutics), an antisense oligonucleotide designed to increase dystrophin, followed by other agents, which include golodirsen, viltolarsen, and casimersen. Continue reading →

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Genetic Therapy Shows Promise for ALS and Dementia in Animal Models – Technology Networks

Posted: Published on February 20th, 2024

Macquarie University neuroscientists have developed a single-dose genetic medicine that has been proven to halt the progression of both motor neurone disease (MND) and frontotemporal dementia (FTD) in mice and may even offer the potential to reverse some of the effects of the fatal diseases. It may also hold opportunities for treating more common forms of dementia, such as Alzheimers disease, which is the second most common cause of death in Australia after heart disease. The new treatment, dubbed CTx1000, targets pathological build-ups of the protein TDP-43 in cells in the brain and spinal cord. Continue reading →

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Cellenkos Approved to Proceed with CK0803 Neurotrophic T regulatory Cell therapy to Treat Second Cohort in ALS Trial – PR Newswire

Posted: Published on February 20th, 2024

HOUSTON, Feb. Continue reading →

Posted in ALS Treatment | Comments Off on Cellenkos Approved to Proceed with CK0803 Neurotrophic T regulatory Cell therapy to Treat Second Cohort in ALS Trial – PR Newswire

Significant Setback in ALS Treatment Development: What’s Next for Neurological Disease Treatments? – Medriva

Posted: Published on February 20th, 2024

A Significant Setback in ALS Treatment Development Denali Therapeutics and Sanofi, two notable names in the pharmaceutical industry, recently suffered a significant setback in their attempt to develop a promising treatment for Amyotrophic Lateral Sclerosis (ALS). The drug, which was in a mid-stage trial, did not meet its primary objective, casting a shadow over the future prospects of this potential treatment option. ALS is a progressive neurological disease that affects nerve cells in the brain and spinal cord, leading to muscle weakness and severe physical disability. Continue reading →

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Amylyx to present ALS treatment developments at MDA conference By Investing.com – Investing.com

Posted: Published on February 20th, 2024

CAMBRIDGE, Mass. - Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) has announced that it will present new research on two of its investigational treatments for amyotrophic lateral sclerosis (ALS) at the upcoming Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in Orlando, Florida. Continue reading →

Posted in ALS Treatment | Comments Off on Amylyx to present ALS treatment developments at MDA conference By Investing.com – Investing.com

There’s AI, and Then There’s AGI: What You Need to Know to Tell the Difference – CNET

Posted: Published on February 20th, 2024

Imagine an AI that doesn't just answer questions like ChatGPT, but can make your morning coffee, do the dishes and care for your elderly parent while you're at work. Continue reading →

Posted in Artificial General Intelligence | Comments Off on There’s AI, and Then There’s AGI: What You Need to Know to Tell the Difference – CNET

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