Study and Editorial on Cannabinoid Hyperemesis Syndrome (CHS) Published in The Journal of Investigative Medicine (JIM)

BEVERLY, Mass., Nov. 30, 2020 /PRNewswire/ — The December issue of The Journal of Investigative Medicine (JIM), the journal of the American Federation for Medical Research (AFMR), is publishing both a study and editorial on the important topic of Cannabinoid Hyperemesis Syndrome (CHS).

The research is entitled Cannabinoid Hyperemesis Syndrome: Definition, Pathophysiology, Clinical Spectrum, Insights into Acute and Long-Term Management.

The Study: Although cannabinoid hyperemesis syndrome (CHS) was first reported more than 15 years ago, it still remains an unfamiliar clinical entity among physicians worldwide. With the legalization of marijuana in many states, CHS will become an increasingly prevalent clinical entity, so educating about CHS is an important goal, particularly for emergency department physicians who generally first encounter these patients.

Authors: Mahesh Gajendran MD, Joshua Sifuentes, MD, Mohammad Bashashati, MD and Richard McCallum, MD of Texas Tech University Health Sciences Center, El Paso.

The editorial, Cannabinoid Hyperemesis Syndrome: The Conundrum Is Here to Stay, is authored by Ron Schey, MD, University of Florida Health, Jacksonville.

Other December highlights include:

  • Review: Advances in the management of atrial fibrillation with a special focus on nonpharmacological approaches to prevent thromboembolism: A review of current recommendations.
    Authors:Harsha S. Nagarajarao, MD, Richard McCallum, MD, Chandra Prakash Ojha, MD, Adriana Camila Mares, MD and Ahmed Ibrahim, MD of Texas Tech University Health Sciences Center El Paso; Malini Riddle, MD of Paul L. Foster School of Medicine, Texas Tech University Health Sciences Center El Paso; Timir Kumar Paul, MD of East Tennessee State University; Vineet Gupta, MD of University of California San Diego; David Alan Baran, MD of Sentara Healthcare; Bharat Ved Prakash, MD of Texas Tech University Health University Health Sciences Center El Paso, Transmountain Campus; Amogh Misra, MD of The University of Texas at Austin; Moeen Abedin, MD and Venkatachalam Mulukutla, MD of University Medical Center of El Paso; Archana Kedar, MD of University of Louisville School of Medicine.
  • Faculty Development & Education:Academic Collaborations with Industry — Lessons for the Future.
    Authors: S. Sethu K. Reddy, MD of Central Michigan University College of Medicine, Mount Pleasant; Shing Chao, MD of College of Medicine, Central Michigan University, Saginaw.

About the Journal of Investigative Medicine: The Journal of Investigative Medicine (JIM) is the official publication of the American Federation for Medical Research. The journal is peer-reviewed and publishes high-quality original articles and reviews in the areas of basic, clinical, and translational medical research.

About the American Federation for Medical Research: The American Federation for Medical Research (AFMR) is an international, multidisciplinary association of scientists engaged in all areas of biomedical and patient-oriented research — from the laboratory to translational to clinical. It works to foster the development of future generations of clinical scientists and investigators through its own initiatives, as well as encouraging public, private, and governmental investment

Read more

Taysha Gene Therapies Receives Rare Pediatric Disease Designation and Orphan Drug Designation for TSHA-104 to Treat SURF1-Associated Leigh Syndrome

Taysha anticipated to submit Investigational New Drug Application for TSHA-104 to FDA in 2021

Rare pediatric disease and orphan drug designations now obtained in multiple pipeline programs, including TSHA-101 for GM2 gangliosidosis, TSHA-102 for Rett syndrome and TSHA-118 for CLN1

Taysha Gene Therapies Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that it has received rare pediatric disease designation and orphan drug designation from the U.S. Food and Drug Administration (FDA) for TSHA-104, an AAV9-based gene therapy in development for SURF1-associated Leigh syndrome. Taysha anticipates it will submit an Investigational New Drug (IND) application to the FDA for TSHA-104 in 2021.

“We have now obtained rare pediatric disease and orphan drug designations in multiple gene therapy programs, which we believe will allow us to work more effectively with the FDA as we advance our broad portfolio,” said RA Session II, President, CEO and Founder of Taysha. “The receipt of these designations highlights the dedication that our team has to advancing our gene therapy pipeline as efficiently and rapidly as possible.”

Leigh syndrome is a severe neurological disorder that usually presents in the first year of life. It is characterized by progressive loss of mental and movement abilities that can result in death within two to three years. Approximately 10-15% of people with Leigh syndrome have a SURF1 mutation.

“Being diagnosed with a mutation in the SURF1 gene is a truly devastating event for families,” said Kasey Woleben, Founder of Cure SURF1 Foundation. “Taysha’s commitment to developing a gene therapy for SURF1 deficiency is greatly welcomed by the patient community and has the potential to save the lives of children afflicted with this progressive disorder.”

Taysha has secured rare pediatric disease designation and orphan drug designation for multiple of its programs, including GM2 gangliosidosis, CLN1, Rett syndrome and now SURF1. In addition to these designations, the company also has fast track status for the CLN1 program.

“SURF1 deficiency is a monogenic mitochondrial disorder and is the most common cause of cytochrome c oxidase deficient Leigh syndrome,” said Steven Gray, Ph.D., Chief Scientific Advisor of Taysha and Associate Professor in the Department of Pediatrics at UT Southwestern. “Obtaining these key designations highlights our commitment to developing a gene therapy for the treatment of SURF1 deficiency.”

The FDA defines a rare pediatric disease as a serious or life-threatening disease in which the disease manifestations primarily affect individuals aged from birth to 18 years. Pediatric diseases recognized as “rare” affect under 200,000 people in the U.S. The Rare Pediatric Disease Priority Review Voucher Program is intended to address the challenges that drug companies face when developing treatments for these unique patient populations. Under this program, companies are eligible to receive a priority review voucher following approval of a product with rare pediatric disease designation if the marketing application submitted for the product satisfies certain

Read more

RNA COVID Vaccines; Down Syndrome Guidelines: It’s TTHealthWatch!

TTHealthWatch is a weekly podcast from Texas Tech. In it, Elizabeth Tracey, director of electronic media for Johns Hopkins Medicine, and Rick Lange, MD, president of the Texas Tech University Health Sciences Center in El Paso, look at the top medical stories of the week. A transcript of the podcast is below the summary.

This week’s topics include two RNA SARS-CoV2 vaccines, an inactivated vaccine, a look at who’s willing to be vaccinated, and guidelines for managing Down syndrome manifestations.

Program notes:

1:06 RNA vaccines

2:00 Similar immune responses young or old

3:00 Inactivated virus

3:10 Vaccine hesitancy

4:10 Vaccine attributes multiple

5:12 Suggests ways to address hesitancy

6:13 Rigorously tested even for an EUA

6:41 An inactivated viral vaccine

7:42 Inactivated vaccines must be made properly

8:44 Not a narrow vaccine

9:12 Guidelines for managing Down syndrome manifestations

10:13 Literature survey

11:12 Congenital heart disease common

12:45 End

Transcript:

Elizabeth Tracey: When a SARS-CoV-2 vaccine comes across the transom, will you take it?

Rick Lange: Report on a promising inactivated whole-virion COVID vaccine.

Elizabeth: What’s the best care for people with Down syndrome?

Rick: And the safety and immunogenicity of two RNA-based COVID vaccines.

Elizabeth: That’s what we’re talking about this week on TT HealthWatch, your weekly look at the medical headlines from Texas Tech University Health Sciences Center in El Paso. I’m Elizabeth Tracey, a Baltimore-based medical journalist.

Rick: And I’m Rick Lange, president of Texas Tech University Health Sciences Center in El Paso, where I’m also dean of the Paul L. Foster School of Medicine.

Elizabeth: And right now in the nation’s hotspot, I would say, relative to battling COVID-19. How’s it going?

Rick: You know, it’s really tough here. I think we have the highest number of cases per capita across the U.S. and increasing. It’s a really tough time in El Paso right now, so some of the stuff we’re talking about is very relevant.

Elizabeth: Yeah. Let’s turn, then, to… why don’t you talk about the first vaccine candidate that you’d like to illustrate?

Rick: Okay. This is a report from Pfizer and a company called BioNTech that is one of those vaccines that’s in phase III trials, so this is the initial report of their phase I and phase II trials. BioNTech and Pfizer launched a coordinated program to compare four RNA-based COVID-19 vaccine candidates. They did it in Germany initially and then brought it to the U.S.

This is a report on two of those RNA-based vaccines and how they decided for one over the other. I’m going to call them B1 and B2, because that’s what they call them. B1 is a RNA vaccine directed towards the receptor-binding domain. They took three of these domains and actually bound them together, which could be more antigenic, and the B2 was they took the whole spike protein.

They tested these in individuals 18 to 55 and those 65 to 85, and they did multiple different doses of these. What they discovered is

Read more

Down syndrome: People with the syndrome face 10 times the risk of death from Covid-19

“This was after adjustment for cardiovascular and pulmonary diseases and care home residence, which our results suggest explained some but not all of the increased risk,” the researchers wrote.

Their analysis involved more than 8 million adults who were part of a coronavirus risk assessment project sponsored by the British government. Of the 8.26 million people in the tracking study, 4,053 had Down syndrome. Of those, 68 people with the disability died and 40% were killed by Covid-19. Seventeen died of pneumonia or pneumonitis and 35% died of other causes.

Those numbers compare with more than 41,000 people without Down syndrome who died, but just 20% died from the coronavirus, 14% from pneumonia or pneumonitis and 65% died of other causes.

Down syndrome is not included in any guidance from the US Centers for Disease Control and Prevention or the UK’s health ministry as a condition that would put people at increased risk for Covdi-19.

“However it is associated with immune dysfunction, congenital heart failure, and pulmonary pathology and, given its prevalence, may be a relevant albeit unconfirmed risk factor for severe COVID-19,” researchers concluded.

A community at risk

National Down Syndrome Society President and CEO Kandi Pickard said her group is grateful that the study has put a focus on the impact of Covid-19 on people with Down syndrome.

“From the beginning of the pandemic, we have been concerned about our community, especially given the complex medical histories of many of our loved ones,” Pickard said. “This recent study confirms our concerns.”

Kids struggle with Covid-19 and its months of aftermath
The society and other Down syndrome organizations worked together to issue the “Q&A on COVID-19 and Down Syndrome” resource guide to help caregivers and others. People with Down syndrome often communicate and understand information in different ways, and they may have trouble understanding social distancing, masking and other ways to prevent infection.

“People with Down syndrome may also have a hard time telling others when they don’t feel well,” according to the coalition. “They may have trouble knowing they have symptoms or how to describe them. For these reasons, they may not raise concerns or seek medical care quickly. Therefore, it is necessary to pay close attention and be watchful.”

Down syndrome is the most common genetic condition diagnosed in the United States every year, according to the CDC, with more than 6,000 babies born with the disability every year. Down syndrome occurs in one in every 700 babies.

Source Article

Read more

Dravet Syndrome Treatment Market | Market to Grow by Over USD 396 Million During 2020-2024

The Dravet syndrome treatment market is set to grow by USD 396.15 million accelerating at a CAGR of over 9%, during the period spanning over 2020-2024. One of the key factors driving growth is the rising strategic alliances. Several pharmaceutical companies are working together to develop drugs for the treatment of Dravet syndrome. Increasing new product launches is a significant trend that will further stimulate market growth. Increasing product launches are making the market more competitive and increasing the availability of drugs for patients.

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20201022005201/en/

Technavio has announced its latest market research report titled Global Dravet Syndrome Treatment Market 2020-2024 (Graphic: Business Wire)

To learn more about the global trends impacting the future of market research, download a free sample now

Parent Market Analysis

Technavio categorizes the Dravet syndrome treatment market as a part of the pharmaceutical market within the overall healthcare industry. The parent, pharmaceuticals market covers products and companies engaged in R&D or production of generic drugs, non-generic drugs, and veterinary drugs.

Growth in the healthcare market will be driven by factors such as increasing life expectancy, improving access to better healthcare in emerging economies, rising prevalence of sedentary lifestyle, rising cases of chronic conditions, and rising number of partnerships between pharmaceutical companies.

Technavio’s in-depth research has all your needs covered as our research reports include all foreseeable market scenarios, including pre- & post-COVID-19 analysis. Download a Free Sample Report on COVID-19 Impacts

Dravet Syndrome Treatment Market: Segmentation by Geography

The report segments the market by Geography: North America, Europe, Asia, and ROW. 44% of the market’s growth will originate from North America during the forecast period. The US and Canada are the key markets for Dravet syndrome treatment in North America.

Dravet Syndrome Treatment Market: Segmentation by Product

The SGDs segment was leading the market in 2019. This report provides an accurate prediction of the contribution of all the segments to the growth of the Dravet syndrome treatment market size.

Dravet Syndrome Treatment Market: Growth Drivers

The market is driven by the rising number of strategic alliances among vendors. Many vendors in the market are partnering with other vendors to develop effective drugs for the treatment of Dravet syndrome. For instance, in September 2018, Supernus Pharmaceuticals Inc. announced its plans to acquire Biscayne Neurotherapeutics (Biscayne), a privately-held company developing a novel treatment for epilepsy. Similarly, in August 2020, Takeda and Ovid completed the Phase II trials of the Dravet syndrome drug and are planning for the Phase III registrational trials. Such developments are expected to boost market growth during the forecast period.

Dravet Syndrome Treatment Market: Challenges to Overcome

Many patients who do not respond to medications are looking for alternative treatment methods to treat Dravet syndrome. Some of the alternative treatment options such as herbs, vitamins, biofeedback, acupuncture, VNS, and keto diet are gaining popularity, which is affecting the growth of market vendors.

Dravet Syndrome Treatment Market: Vendor landscape

This report provides information on

Read more

What it’s like to live with stiff person syndrome

Jane Lees, 58, lives in Indianapolis and used to work in media. Last year, she suddenly lost a lot of weight and began experiencing extreme muscle pain. She learned a rare condition was behind it. She told her story to TODAY to encourage others to advocate for their health.

Everyone likes to think of themselves as 1 in a million and last year I learned I truly am. After a series of symptoms that ranged from mild to worrisome, I learned that I have stiff person syndrome, a neurological condition that affects 1 in a million people, mostly women. Having a unique condition has been tough, but I have spent the time trying to understand who I am now that I live with this rare illness.

I love to cook, but now I need to sit down every few minutes to build up my strength to continue. (Courtesy Jane Lees)
I love to cook, but now I need to sit down every few minutes to build up my strength to continue. (Courtesy Jane Lees)

I first noticed something wrong when I joined my daughters for vacation last year in Florida. I struggled to keep up with them. But the real wake-up call came when I visited my college roommate and she was shocked by my appearance — I weighed about 87 pounds after suddenly dropping 20 pounds in a month without explanation.

Then in early May, I woke one morning and realized I could no longer ignore what was happening. I felt like I had the flu, but I had received a flu vaccination. Did I somehow get the flu from the vaccine? Doctors felt somewhat puzzled by my condition, but found that I had an infection and treated me for it.

For months, doctors didn't know what was causing my extreme muscle pain, fatigue and stomach upset. Finally a med student suggested stiff person syndrome. (Courtesy Jane Lees)
For months, doctors didn’t know what was causing my extreme muscle pain, fatigue and stomach upset. Finally a med student suggested stiff person syndrome. (Courtesy Jane Lees)

I never completely recovered. The left side of my body felt so tight that I’d ask my daughters to help stretch it. I struggled to walk and I felt unsteady on my feet. My body was so stiff and weak that I felt like I did not have the strength for basic tasks. I was in constant pain. By September I returned to the hospital and was diagnosed with Type 1 diabetes. I felt relief and imagined feeling better soon, but it never happened. I still had crippling pain, stiffness and weakness.

The team at Indiana University Health ran every test they could think of trying to uncover the reason why my muscles felt so tight, but still felt at a loss about what was behind my pain.

Then a med student mentioned something that the doctors and nurses had never heard of — stiff person syndrome.

Because stiff person syndrome compromises my immunity, I can't see many people these days. Though I still appreciate time with my family. (Courtesy Jane Lees)
Because stiff person syndrome compromises my immunity, I can’t see many people these days. Though I still appreciate time with my family. (Courtesy Jane Lees)

At first I felt overjoyed there was a name for what I was experiencing. But that turned to worry when doctors admitted that they don’t really understand how to treat this

Read more

Treating Facial Tics and Tourette’s Syndrome With Alternative Medicine

Tics may be due to nutritional deficiencies, genetic conditions, Tourette syndrome, immune system malfunction, allergies, or stress. Stress and anxiety can increase the frequency of facial tics. Emotional trauma can cause tics which can disappear when the emotional disorder is treated. Tics due to emotional trauma or stress can improve or disappear with hypnotherapy, Emotional Freedom Technique (EFT), energetic therapy, Neuro-Linguistic Programming (NLP) yoga, Tai Chi, music therapy, homeopathy, Interactive Metronome, cognitive behavioral therapy, HEMI Sync, acupuncture, massage, and scalp acupuncture. Secondary mood disorders can worsen the severity of tics.

Treating and healing facial tics or Tourette’s syndrome with ordinary amino acids is the basis for Orthomolecular Medicine. Large doses of naturally found proteins called amino acids can repair the imbalance in the brain and repair its malfunction. These amino acids are affordable and are used in therapeutic doses much larger than those levels normally found in food. The concept of orthomolecular medicine is based upon the use of very large doses of vitamins, minerals, amino acids, or botanical extracts for the cellular repair and enhancement of normal brain activities and motor activities.

Lithium mineral salt may help this patient if there is Tourette’s syndrome, but it would not help for most other types of facial tics. Botanicals that may treat tics and reduce tics and spasms include kava, skullcap, valerian, St John’s Wort, peppermint, black cohosh, dragon bone, prunella, rose hips, Go Teng, Tian Men Dong, Bai Shao, Yin Chen Hao. Orthomolecular medicine uses includes magnesium, zinc, calcium, B Vitamins, and chromium along with the botanicals to support recovery.

Orthomolecular medicine uses large therapeutic doses of carnitine, tryptophan, taurine, GABA, and 5-HTP. Theanine, GABA, and 5-HTP can work well for tics caused by stress. These amino acids must be used long- term for Tourette’s syndrome and you may want to consider IV therapy in the worst cases for at least nine months.

This patient may have severe allergies and benefit from the elimination of genetically modified foods. The diet should be altered to exclude unsaturated fats, caffeine, nicotine, artificial sweeteners, food additives, food dyes, alcohol, and high fat meats. Try to eat free range chicken, organic meat, organic or locally produced eggs, and limit red meat to twice per week. You should always eat breakfast relatively high in protein. This may include whey protein, almond milk, or soy protein. Walnuts, yogurt, almonds, pumpkin seeds, sunflower seeds, and pecans are great snack foods ideas. Remember that corn is frequently a genetically modified food. Popcorn can be a good snack food if you are certain that it is not genetically modified. Organic or locally grown vegetables and fruits are the best health options. Avoid foods that are often contaminated with pesticides or heavy metals. Plant fruit trees in your yard for more fresh fruit.

Source Article

Read more

What You Need To Know About Functional Medicine & Treating Irritable Bowel Syndrome

You've got questions about natural treatments for IBS, and we have answers.

We treat a lot of IBS cases in practice in our Longmont and Denver, CO offices. Often, we see patients who have run the gauntlet with chronic GI pain, gas, bloating, constipation and diarrhea (or a combination of all of these), who have seen every doctor and taken all the prescription medications they've been recommended, and yet, they still feel miserable and their symptoms have not subsided. Or, they get some temporary relief, only to find new and (uncomfortable) triggers resulting in more flare ups.

We practice Functional Medicine. We take a different approach to treating gut issues. Especially Irritable Bowel Syndrome, because it affects people so differently, yet it's disease processes are often similar for most sufferers. In Functional Medicine, we do not automatically assume IBS is solely a "gut issue" because we know that IBS is often the symptomatic outcome of a combination of issues.

One of the first things we will recommend is blood work or other diagnostic testing (depending on your specific situation). Now, that may be met with a bit of hesitation at first as you say to yourself, "well, I've had blood work done already and they told me there's nothing wrong! Everything checked out as 'normal.'" Depending on the specific training of whom exactly is looking at and interpreting the results of these tests can mean a world of difference between continued recurrences of IBS, and the relief you're seeking. We say this because not all tests are created equally and viewing their results through the lens of Functional Blood Chemistry (which uses a different set of reference ranges) can detect whether the symptoms you experience are from infection, stress, a food sensitivity or a combination of many factors. Many men and women with IBS suffer from:

  • SIBO (Small Intestine Bacterial Overgrowth)
  • Parasites
  • H. pylori
  • Yeast overgrowth
  • Food sensitivities

It is imperative then to treat the root cause (s) of the IBS in order to control the symptoms. We generally like to treat IBS as a metabolic condition, because all of the body's systems must be brought back into harmony before any real, quantifiable, long term relief can begin. In addition to treating determinable IBS protagonists like those above, a "typical" Functional Medicine IBS protocol should also include …

A Metabolic cleanse heavily focusing on the Hepatic system. Clearing the liver, pancreas and gallbladder of sludge and toxins helps support those organs in order for them to work most efficiently. Not to mention, when these organs are fatigued, they cannot clear hormones as intended or help in digestion properly.

Next we will look at the patient's diet. We will either have an idea of ​​foods they need to permanently avoid from the results of their food sensitivity test (s), or we will recommend an elimination diet to find offending items. In our practice we strongly recommend food sensitivity testing through a lab called Cyrex. They have many arrays and can test …

Read more