Weekly Roundup – August 19, 2023
Welcome to our Healthcare IT Today Weekly Roundup. Each week, we’ll be providing a look back at the articles we posted and why they’re important to the healthcare IT community. We hope this gives you a chance to catch up on anything you may have missed during the week.
Helping Children With Gamified Interactive Physiotherapy. Psychiatrist Dr. Geoff Frost struggled to encourage pediatric patients with spinal muscular atrophy to do their necessary exercises. As he explained to Colin Hung, that’s why Raft Digital Therapeutics created a game called Cloud Bazaar to captures a child’s movement as a way to advance...
Source: EMR and HIPAA - August 19, 2023 Category: Information Technology Authors: Brian Eastwood Tags: Healthcare IT Healthcare IT Today Weekly Roundup Source Type: blogs
Helping Kids with SMA Win with a Physiotherapy Game
Physiotherapy is hard. It is even harder for children who get bored and tire quickly from the repetitive motions that doctors prescribe for them. Raft Digital Therapeutics (Raft) created an interactive video game to help children with a rare degenerative neuromuscular disease – Spinal Muscular Atrophy (SMA) – to maintain their physical abilities, improve outcomes, and promote a higher quality of life. Their game has found success with children, parents, and clinicians.
Healthcare IT Today had the opportunity to sit down with Dr. Geoff Frost, CEO of Raft and practicing physiatrist, to find out about their game, their re...
Source: EMR and HIPAA - August 17, 2023 Category: Information Technology Authors: Colin Hung Tags: Clinical Communication and Patient Experience Health IT Company Healthcare IT Hospital - Health System Telemedicine and Remote Monitoring Cloud Bazaar Collision 2023 Collision Conference Digital Therapeutics Dr. Geoff Frost gamificia Source Type: blogs
How Technology Can Help Overcome Barriers to Getting Effective Therapies into Patients with Ultra Rare Disorders
The following is a guest article by Dr. Zach Landman, Co-Founder of Moonshots for Unicorns.
As a physician who trained at UCSF, Harvard, and Stanford, I assumed that when my youngest daughter, Lucy – at 10-months old – was diagnosed with an ultra-rare genetic disorder of glycosylation called PGAP3, the answers would reside within a hospital or academic laboratory.
Unfortunately, my pediatrician wife and I were told that our smiling, seemingly healthy babbling 10-month-old baby would likely never walk normally, never talk, and was likely to develop severe and refractory seizures at some point in her childhood. ...
Source: EMR and HIPAA - September 27, 2022 Category: Information Technology Authors: Guest Author Tags: Clinical Healthcare IT AAV9 ASO CRISPR Dr. Zach Landman epalrestat Gene Therapy Invitae Patient Stories Patients Perlara PGAP3 PMM2 PRAX-222 Rare Diseases SCN2A SMA-1 Source Type: blogs
Fight Aging! Newsletter, June 27th 2022
In conclusion, this study confirms that innate immune training can be induced in aging healthy individuals as well as critically ill sepsis patients. We found that innate immune training can be induced regardless of age and there was no substantive difference in the immune trained phenotype as a function of age. We employed β-glucan as our immune training stimulus. The ability of glucan to induce the trained phenotype suggests that it may be possible to pharmacologically induce the immune trained phenotype in aging human immunocytes.
Sitting Time Correlates with Mortality Risk
https://www.fightaging.org/archi...
Source: Fight Aging! - June 26, 2022 Category: Research Authors: Reason Tags: Newsletters Source Type: blogs
Phosphorylated TDP-43 in Muscle as a Biomarker for ALS
TDP-43 is one of the proteins more recently discovered to become phosphorylated and form harmful aggregates in aged tissues. These aggregates are connected to a range of neurodegenerative conditions, including amyotrophic lateral sclerosis (ALS). Researchers here discuss a novel way to use TDP-43 as a biomarker for the early onset of ALS. Being able to quantify the progression of neurodegenerative conditions in their early stages is a necessary part of the development of effective means of prevention.
Clinicians diagnose ALS based on deteriorating motor function, such as weakness in the arms or legs or difficulty ...
Source: Fight Aging! - June 20, 2022 Category: Research Authors: Reason Tags: Daily News Source Type: blogs
ALS2 Funding Opportunity: Fundamental Research in Underlying Causes of Amyotrophic Lateral Sclerosis
We’re pleased to announce our participation in the Accelerating Leading-edge Science in ALS (ALS2) program (NOT-RM-20-019). ALS2 is a $25 million NIH Common Fund initiative to spur innovative research into the basic biology of amyotrophic lateral sclerosis (ALS) through the NIH Director’s Transformative Research Award. NIGMS encourages our community of basic scientists working on relevant processes to consider applying for this opportunity.
ALS is a devastating disease with no known cure. The development of effective therapeutics can benefit tremendously from basic ALS research that 1.) tests highly novel concepts, ...
Source: NIGMS Feedback Loop Blog - National Institute of General Medical Sciences - June 30, 2020 Category: Biomedical Science Authors: Chrissa Chverchko Tags: Funding Opportunities NIH Common Fund Source Type: blogs
Never before has it been more exciting and important to be a neurologist
What are the current trends and hot topics in neurology?
The increasing insights into the genetics and molecular bases of neurological disorders open new perspectives for specific and personalized treatments. This is evidenced most dramatically by the antisense therapy for spinal muscular atrophy. Promising neurological research currently targets neurodegenerative diseases such as amyotrophic lateral sclerosis, Parkinson’s disease, Duchenne muscular atrophy, or Huntington’s disease. Likewise, brain-machine interfaces and neuroprosthetics offer huge potential. Never before has it been more important and more exciting to...
Source: BioMed Central Blog - December 9, 2019 Category: General Medicine Authors: Victoria Hentschke Tags: Uncategorized Source Type: blogs
How Much Is Life Worth?
Multivitamins, drugs, gene therapies, human skin, heart, eyeballs, kidneys, entire dead bodies – everything comes with a price tag. Putting aside the moral questions of why and how come that the capitalist market priced even our body parts and health, we asked the question of how much is life worth: what is the maximum that you would/should pay for a life-saving drug? How high is too high a cost if a drug can save 200-300 babies a year from debilitating illness or death? And ultimately, does the pricing of new technologies, especially gene therapies, enable to fulfill their promise?
There’s a
price for everything: ...
Source: The Medical Futurist - August 31, 2019 Category: Information Technology Authors: nora Tags: Bioethics Biotechnology Future of Pharma Genomics cost daraprim drug drug price Gene gene therapy genetics insulin life medication pricing policy rare disease rare disorder Source Type: blogs
The rule of rescue
I ' ve written about the rule of rescue quite a bit, but not recently.Now the approval of a new treatment for the genetic disease Spinal Muscular Atrophy is an occasion to revisit the topic. Some of the best discussion of this is in theNew York Times, but it ' s behind a paywall for many of you so I ' m providing alternative links.SMA can have slow onset, but for many it means death in early childhood without treatment. There is a previously approved treatment, which costs $375,000/year. The newly approved treatment is thought to require only a one-time infusion, which may provide life-long benefit, but it costs $2.1 milli...
Source: Stayin' Alive - August 26, 2019 Category: American Health Source Type: blogs
The ethics behind the world ’s most expensive medication
With the recent FDA approval, Zolgensma became the world’s most expensive medication. Priced at $2.125 million per patient, the one-dose gene therapy is a potential life-saver for children with spinal muscular atrophy (SMA). Now, the treatment is at the center of an intensifying debate over the rising price of medications. Industry watchdogs ar e outraged. They say Zolgensma […]Find jobs at Careers by KevinMD.com. Search thousands of physician, PA, NP, and CRNA jobs now. Learn more. (Source: Kevin, M.D. - Medical Weblog)
Source: Kevin, M.D. - Medical Weblog - June 14, 2019 Category: General Medicine Authors: < span itemprop="author" > < a href="https://www.kevinmd.com/blog/post-author/robert-pearl" rel="tag" > Robert Pearl, MD < /a > < /span > Tags: Meds Medications Neurology Source Type: blogs
You can save your baby for $2 million. Can we afford to do this?
You can save your baby for $2 million. Can you afford to do this? If you cannot, should we all pay? Spinal muscular atrophy (SMA) is a rare genetic disease affecting the part of the nervous system that controls voluntary muscle movement. All types of SMA result from a single known cause, a deficiency of […]Find jobs at Careers by KevinMD.com. Search thousands of physician, PA, NP, and CRNA jobs now. Learn more. (Source: Kevin, M.D. - Medical Weblog)
Source: Kevin, M.D. - Medical Weblog - May 31, 2019 Category: General Medicine Authors: < span itemprop="author" > < a href="https://www.kevinmd.com/blog/post-author/edward-hoffer" rel="tag" > Edward Hoffer, MD < /a > < /span > Tags: Meds Genetics Medications Source Type: blogs
Looking to the Future of Neuro Devices: Exclusive Interview with Alcyone Lifesciences CEO PJ Anand
Alcyone Lifesciences is a medical technology company based out of Lowell, MA, which specializes in central nervous system (CNS) drug delivery platforms for targeted infusions of the brain or spinal cord. Their Thecaflex DRx System was recently awarded Breakthrough Device designation by the FDA, for spinal infusion of therapeutic medications.
Medgadget had the opportunity to speak with Alcyone Lifesciences CEO, PJ Anand, about his inspiration for cutting edge medical technology and the future direction of neurological devices.
Kurt Yaeger, Medgadget: Great to talk to you, PJ. Please first give us a sense of your back...
Source: Medgadget - May 7, 2019 Category: Medical Devices Authors: Kurt Yaeger Tags: Exclusive Neurology Neurosurgery Pain Management Psychiatry Source Type: blogs
How should we measure quality of life impact in rare disease? Recent learnings in spinal muscular atrophy
Office of Health Economics -The measurement of quality of life in the context of spinal muscular atrophy (SMA) is challenging. This is because the disease is experienced by children and is rare, which makes data collection difficult. This briefing reports on a symposium that outlined some lessons that can be learnt from the SMA context that might be more widely applicable to other rare diseases.BriefingMore detail (Source: Health Management Specialist Library)
Source: Health Management Specialist Library - April 1, 2019 Category: UK Health Authors: The King ' s Fund Information & Knowledge Service Tags: Quality of care and clinical outcomes Source Type: blogs
New CNS Drug Delivery System by Alcyone Lifesciences Given Breakthrough Designation by FDA
Alcyone Lifesciences recently obtained Breakthrough Device Designation from the FDA for their novel implantable intrathecal bolus drug delivery catheter and port system, the ThecaFlex DRx System. The system is intended for use in conditions that require prolonged medication administration directly into the cerebrospinal fluid (CSF), such as some cancers and neurodegenerative diseases. Currently, these patients require frequent, repeated spinal tap procedures in order to obtain correct dosing of medication into the central nervous system. Surgical implantation of the Alcyone ThecaFlex DRx system may obviate the need for thi...
Source: Medgadget - April 1, 2019 Category: Medical Devices Authors: Kurt Yaeger Tags: Neurology Neurosurgery Pain Management Radiology Source Type: blogs
