motor neurone disease

PharmAust (ASX: PAA) chief executive officer Michael Thurn joins Small Caps to discuss the company's ongoing success with its investigation into its lead treatment candidate monepantel (MPL).

PharmAust is focused on repurposing MPL for the treatment of human neurodegenerative disorders such as motor neurone disease (MND) / amyotrophic lateral sclerosis (ALS).

New top-line Phase 1 MEND study results have found that PharmAust has taken a significant step towards helping people diagnosed with this rare and incurable disease.

Notably, new tests have found that MPL displays a superior safety, tolerability to the leading FDA approved drug Relyvrio.

Preliminary efficacy data also found a 58% reduction in the rate of disease progression for high dose Cohort 2 patients using the FDA primary efficacy endpoint.

The result has demonstrated the potential to provide meaningful clinical benefit to people living with MND/ALS.

PharmAust also recently created a “dream team” of scientists including internationally renowned experts in MND/ALS drug discovery and clinical development who will provide PharmAust with strategic guidance in the development of MPL

Articles:
https://smallcaps.com.au/pharmaust-monepantel-study-shows-slowdown-mnd-progression/
https://smallcaps.com.au/pharmaust-world-class-scientific-advisory-board-fight-against-neurodegenerative-diseases/
https://smallcaps.com.au/pharmaust-monepantel-study-mnd-als-treatment-fast-track-fda-meeting/
https://smallcaps.com.au/pharmaust-begins-extended-study-mnd-treatment-successful-trial/

For more information on PharmAust:
https://smallcaps.com.au/stocks/asx-paa/

See omnystudio.com/listener for privacy information.

PharmAust (ASX: PAA) chief executive officer Michael Thurn joins Small Caps to discuss his appointment to the company and recent trial developments.

The company recently released interim analysis of data from a Phase 1 clinical trial of its lead drug candidate monepantel (MPL) to treat motor neurone disease/amyotrophic lateral sclerosis (MND/ALS).

Results from an interim analysis of the trial indicated that neurofilament light chain (Nfl) protein concentrations in the plasma of participants in its Phase 1 clinical trial do not increase following MPL treatment – a positive sign.

PharmAust hopes that MPL could eventually receive orphan drug designation by the Therapeutics Goods Administration (TGA) and US Food and Drug Administration (FDA) for motor neurone disease, which affects over 350,000 people globally and kills more than 100,000 people yearly.

Article:
https://smallcaps.com.au/pharmaust-names-highly-experienced-biotech-specialist-chief-executive-officer/
https://smallcaps.com.au/pharmaust-interim-phase-1-data-mpl-inhibit-motor-neurone-disease/

For more information on PharmAust:
https://smallcaps.com.au/stocks/asx-paa/ 

See omnystudio.com/listener for privacy information.

One expert claims salmon farming is changing ecosystems to the detriment of wildlife and humans. 

There's a scientific story behind Dame Edna's famous Gladioli, and it involves one of Australia's top botanists. 

In this episode, hosts Karl & Craig sit down with MND Fundraiser and Lifelong Wigan fan Graham Berry and Director of Rugby at the NEW Ravens RLFC, Jason Spafford.

👉 Graham Berry - Born in Wigan, Graham was destined to be involved in Rugby league from a young age. Graham talks about finding touch RL in Telford after relocating to the Midlands and eventually going on to raise over £7000 for the MND Association through challenging ultra-running events.

Graham is currently in training for the Trans-Pennine Ultra - a 91-mile run from the DW Stadium to Headingley in Leeds.

↪ MND Link
↪ Fundraising Link

👉 Jason Spafford - Jason has been heavily involved in the development of the Rugby League in the Midlands since as early as 1996 and is now the Director of Rugby at the NEW Ravens club in Redditch. Born in Kent, Jason found Rugby League after a career-ending boxing injury and was immediately hooked.

↪ New Ravens
↪ Follow Jason on Instagram

SPONSOR: This episode was sponsored by Pot Leadle - Digitial Marketing & SEO Specialists in Cheshire

A new study indicates that there is a link between playing rugby professionally and contracting neuro-degenerative diseases later in life.

The study compared former Scottish national rugby union players with the general public and confirmed that the chance of contracting a neuro-degenerative condition was 15 times higher.

Professor in Human Performance at AUT, Patria Hume is here to explain the impact of the study and what factors may have been overlooked in the process of conducting the research.

LISTEN ABOVE

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A new study has just been released confirming that professional rugby players are at a higher risk of contracting head-based conditions, including dementia and motor neuron disease.

D'Arcy Waldegrave, host of Sportstalk, is here to discuss the study and the concerning implications involved.

LISTEN ABOVE 

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When something goes wrong with a brain we can’t just get under the hood and poke around. So how do we figure out what’s going wrong? Well sometimes the answer to that question involves watching monkeys watch TV, so we’re taking a trip to a monkey cinema, and along the way learning about the different ways to see inside your head and find faulty wiring. 

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• Twitter: @marblespod
• Instagram:  @marblespod

Peter Chambers is an inspiring Aussie who, whilst living with the incurable Motor Neurone Disease, has found a reason to be and is living a wonderful life. From deep within he has found peace in the storm and purpose in his adversity and  is now on a mission to make the most of every second, enjoy the simple things in life and make a positive difference in the lives of others.

Peter's first symptoms in late 2017 to early 2018 led to a spinal fusion operation in May 2018. After the operation, his shoulders lost muscle rapidly and, whilst he gradually regained some use in his left arm, his left hand was severely weakened and he has never regained any more than about 10% of movement in his right arm. Neurologists, at the end of 2019, finally agreed the diagnosis was MND. He was diagnosed the Arm Flail version of MND, which lead to his arms losing all muscle first, then other muscles to follow.

Rather than fall into victim mode, Peter chose to find the positives in his situation, to live and love every moment and to fuel his purpose. He now devotes his time and energy to helping promote awareness of the disease and to be a voice of advocacy. I love his attitude towards life and Peter Chambers is a man who will touch your heart and your life. This is a wonderfully inspiring conversation that I encourage you to listen to.

Peter can contacted via his email or through LinkedIn.

Click here to donate to  MND Victoria

My website is www.andrewjobling.com.au

In this episode I cover motor neurone disease.

If you want to follow along with written notes on motor neurone disease go to https://zerotofinals.com/medicine/neurology/mnd/ or the neurology section in the Zero to Finals medicine book.

This episode covers pathophysiology, presentation, types, investigations, diagnosis and management of motor neurone disease. 

The audio in the episode was expertly edited by Harry Watchman.

From the moment of his diagnosis with ALS, Bruce Kramer began writing — openly, deeply, and spiritually — about his struggle, as he puts it, to live while dying. He died on March 23, 2015, while we were in production on this show. His words hold abiding joy and beauty, and reveal an unexpected view opened by this disease.

Bruce Kramer was the creator of “The Dis Ease Diary” a blog about his life with ALS and “We Know How This Ends: Living While Dying.” He was the Dean of the College of Education, Leadership and Counseling at the University of St. Thomas, where he served on the faculty for over 19 years. He was a passionate music lover and was a choir conductor for most of his adult life. This interview is edited and produced with music and other features in the On Being episode “Bruce Kramer — Forgiving the Body: Life with ALS.” Find more at onbeing.org.

Highlights from the March issue with a focus on neurodegenerative disease.

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October issue highlights, including cilostazol compared with aspirin for secondary stroke prevention.

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