`Patients who received 70 mg of zorevunersen..followed by up to 45 mg in the extension studies, had a median change from baseline in convulsive-seizure frequency ranging from −58.82% to −90.91% across 1-month intervals during the first 20 months of the extension studies. The data supported improvements in overall clinical status, quality of life, and adaptive behavior with continued treatment for up to 36 months in the extension studies.`

https://www.nejm.org/doi/10.1056/NEJMoa2506295

#Dravet #epilepsy

Il Fatto Quotidiano: Un “cerotto genetico” contro la Sindrome di Dravet, così un farmaco ha ridotto i sintomi

Un oligonucleotide antisenso, definito come “cerotto genetico”, ha mostrato risultati promettenti nel trattamento della sindrome di Dravet, una forma rara e grave di epilessia congenita con poche opzioni terapeutiche. I dati provengono da studi clinici di fase 1/2a condotti presso l’Ann & Robert H. Lurie Children’s Hospital di Chicago e pubblicati sul prestigiosissima rivista New England Journal of Medicine. La sindrome di Dravet, che colpisce bambini tra i 2 e i 18 anni, è caratterizzata da convulsioni, deficit cognitivi, disturbi della comunicazione e del comportamento, disfunzioni motorie, ritardi della crescita e tratti autistici. La malattia è legata a mutazioni nel gene Scn1a, che porta alla produzione solo parziale della proteina del canale del sodio essenziale per la funzione neuronale.
Il farmaco sperimentale, zorevunersen, viene somministrato tramite puntura lombare e funziona bloccando una sequenza genetica chiamata “esone velenoso”, che normalmente riduce la quantità di proteina Scn1a sana prodotta. In questo modo, zorevunersen aumenta la sintesi della proteina funzionale, compensando il difetto genetico. I due studi multicentrici, condotti negli Stati Uniti e nel Regno Unito, hanno coinvolto 81 pazienti. Coloro che hanno ricevuto due o tre dosi di zorevunersen hanno mostrato una riduzione delle crisi motorie di quasi l’85% a tre mesi e del 73% a sei mesi dalla somministrazione. Oltre alla riduzione delle crisi, i pazienti hanno mostrato miglioramenti significativi in linguaggio, comportamento e qualità della vita, senza evidenti problemi di sicurezza o tollerabilità.
Un ragazzo di 12 anni partecipante alla sperimentazione, ha sperimentato un miglioramento notevole: “La sua qualità di vita è migliorata tanto che ora può svolgere più attività con i coetanei”, ha riferito la madre. Attualmente è in corso uno studio di fase 3 in doppio cieco, controllato con placebo, che mira a confermare i risultati preliminari e a valutare l’efficacia a lungo termine di zorevunersen nella popolazione pediatrica affetta da sindrome di Dravet.
L'articolo Un “cerotto genetico” contro la Sindrome di Dravet, così un farmaco ha ridotto i sintomi proviene da Il Fatto Quotidiano.

A “genetic patch” against Dravet Syndrome, so a drug has reduced the symptoms.

An antisense oligonucleotide, defined as a “genetic patch,” has shown promising results in the treatment of Dravet syndrome, a rare and severe form of congenital epilepsy with few therapeutic options. The data comes from Phase 1/2a clinical studies conducted at the Ann & Robert H. Lurie Children’s Hospital of Chicago and published in the prestigious New England Journal of Medicine. Dravet syndrome, which affects children between 2 and 18 years old, is characterized by seizures, cognitive deficits, communication and behavioral disorders, motor dysfunction, growth retardation, and autistic traits. The disease is linked to mutations in the Scn1a gene, which leads to the partial production of the sodium channel protein essential for neuronal function.

The experimental drug, zorevunersen, is administered via lumbar puncture and works by blocking a genetic sequence called the “toxic exon,” which normally reduces the amount of healthy Scn1a protein produced. In this way, zorevunersen increases the synthesis of the functional protein, compensating for the genetic defect. The two multi-center studies, conducted in the United States and the United Kingdom, involved 81 patients. Those who received two or three doses of zorevunersen showed an 85% reduction in motor seizures at three months and a 73% reduction at six months from administration. In addition to reducing seizures, patients showed significant improvements in language, behavior, and quality of life, without apparent safety or tolerability issues.

A 12-year-old participant in the trial, reported a remarkable improvement: “Her quality of life has improved so much that now she can do more activities with her peers,” reported the mother. Currently, a Phase 3 double-blind, placebo-controlled study is underway, aiming to confirm the preliminary results and assess the long-term efficacy of zorevunersen in the pediatric population affected by Dravet syndrome.

The article “A ‘Genetic Patch’ Against Dravet Syndrome, So a Drug Has Reduced Symptoms” is from Il Fatto Quotidiano.

#DravetSyndrome #Dravet #Scn1a #theUnitedStates #theUnitedKingdom #IlFattoQuotidiano

https://www.ilfattoquotidiano.it/2026/03/05/sindrome-dravet-cerotto-genetico-epilessia-notizie/8313257/

"Is Kaneh-Bosm, Natures Multipurpose Pharmaceutical... " Part 2 of 2 –
Medical and Scientific:
⚠️ CW: Cannabis
Click here 🧠 to expand.

Alzheimer's and Dementia:

In a previous post, I highlighted the medical research of two renowned cannabis experts, Dr. Raphael Mechoulam and Dr. Dedi Meiri. Dr. Meiri's recent studies proclaiming the benefits and safety of treating Alzheimer's with the plant, seems to be mirrored by many other findings. Dr. Mechoulam has been studying cannabis for decades and has published countless results regarding the herb's healing effects on a wide array of health issues and IDs (invisible disabilities). "Cannabis Therapeutics in the Future of Neurology" in the pub med NLB link, speaks of the efficacy and safety track record of Kaneh Bosm, echoing the conclusions of these "experts".

TBI, Brain Injury, CTE and Others:

That study, and others below, examine "the intriguing promise that recent discoveries regarding cannabis-based medicines offer to neurological therapeutics..." Five "intractable conditions" labeled as being "recalcitrant to therapeutic success" were looked at. Numerous THC, CBD and other cannabis compounds were explored for treating Alzheimer's, epilepsy, Parkinson's, brain tumors and TBI/CTE (traumatic brain injury and chronic traumatic encephalopathy). The seeming ability of Cannabis "Botanicals" to interact with, and "harness"* the endocannabinoid system, was also noted in the studies.

The human body is equipped with, what is referred to as the "endocannabinoid system". One of the major parts of this system being, a variety of "endogenous neurotransmitters" that, as a group are referred to as "endocannabinoids". The other major components of this system are two known cannabinoid receptors, CB1R and CB2R. A simplified explanation I once heard, made a comparison: "as the thyroid gland is to the endocrine system, CB receptors are to the endocannabinoid system". When cannabinoids from the plant are introduced, a sort of "activation" of the CB1R and CB2R receptors in the body occurs. Some feel this is the natural health benefits of "Natures Pharmaceutical", working wellness in the body.

"Cannabis Therapeutics in the Future of Neurology" (linked below) reports on "shared pathological process" and the "plausibility" that, the botanical cannabis compounds tested, were responsible for "interventions that harness the endocannabinoid mechanisms". Considering concerns about safety and the damaging side effects of chemical pharmaceuticals, the herb that some have called "Nerve Grease", can bring safe and controlled relief to many with a variety of IDs and other conditions. Just as Dr. Meiri, and the years of Dr. Mechoulam's studies have indicated, the report on pub med.gov states:

1) "Current basic science and clinical investigations support the safety and efficacy of such interventions in treatment of these currently...".
2) "While cannabis-based medicines have demonstrated safety, efficacy and consistency sufficient for regulatory approval in spasticity in multiple sclerosis (MS), and in Dravet and Lennox-Gastaut Syndromes (LGS), many therapeutic challenges remain".

Brain injury (TBI/ABI) and PTSD – My View from the inside:

According to the "Journal of Neuroinflammation": "...the only treatment options for TBI include symptom management and cognitive therapies. There are currently no effective pharmaceutical treatment options to improve outcomes for TBI of any severity despite great clinical need".
As one with both TBI and ABI, I am well aware of the truth in that statement. I have always been "recalcitrant" to the prescription of pharmaceutical "relief", because of my brothers opioid addiction, that ultimately took his life. They were also very ineffective when I did try them. I knew what worked to help my whole being and I was comfortable with Kaneh Botanical intervention as treatment, though others were not.

The varied array of TBI affects include: cognitive impairment, trouble focusing, appetite repulsion, nausea, avoidance/social anxiety, irritability, moodiness, insomnia, tremors, communication limitations, aphasia, memory issues, headaches, inflammation and sensitivity to sound and light. The average life expectancy of people with TBI is 54 years and many are susceptible to early onset Alzheimer's.

I have certainly experienced a legion of these and a number of other symptoms. Early on, I had to relearn things like making coffee, because filling the machine, finding the filter, dispensing the coffee etc., was too many steps, and once took me over two hours. At times, I tremored and stuttered, having the same headache every day, for seven consecutive years. Effects of TBI are not always noticeable immediately following an accident but, can develop over the course of time. About a year after my first accident, I had a few occurrences while driving, of momentarily forgetting where I was going, or what road I was on. I was going home on a familiar thoroughfare. I wasn't fearful, seeming to know it would pass and I'd recognize where I was but, in my mind, I was lost for a few minutes.

When able, I also began spending countless hours researching brain injury, neurology and cannabis as medicine. I found Dr. Mechoulam's work, which resonated with some of my own observations about the relief cannabis provided. After being sidelined by brain injury and other disabilities, I've performed a few "clinical trials" of my own, so to speak. Contrary to some opinions about cannabis, the brain can be affected in many positive ways. 20 years later, considering my own brain's "regeneration" and Mechoulam's and other reports on the neuroprotective antioxidant qualities of the flower, there's been no doubt, cannabis has played a big role in my cognitive, emotional and physical recovery.

From other supporting research below: "Pre-clinical trial TBI research suggests that cannabinoids have neuroprotective and psychotherapeutic properties".
Dr. Staci Gruber, an associate professor of psychiatry at Harvard Medical School, tested patients using cannabis as treatment. A number of control assessments were performed before the patients took cannabis and at various intervals in the months that followed. Dr. Gruber reports a marked improvement in "cognitive tasks requiring executive function", after only a few months. Gruber said that patients may be thinking more clearly because of symptom relief and less reliance on pharmaceuticals, like opioids.

In TBI related studies, Biomedical researcher Dr. David Plurad found cannabis to be a "natural insulator for traumatized brains" and said it can "restore normal endocannabinoid function". His study states that the introduction of cannabis after a traumatic event can prevent PTSD like symptoms, lending support to the findings of Dr. Mechoulam. Dr. Plurad's report said cannabinoid compounds can help stabilize mood, ease depression and "are better at treating PTSD than SSRI antidepressants, which bring a host of negative side effects, including violent tendencies".

When I started an outline for this article, I began with a list of benefits that I was aware of or had personally experienced. In researching, I found an almost identical list among the NLM pub med links below that also contains the cannabis compounds found to be effective for each listing. I'll be posting both lists in the comments section later.

If I were asked for my "conclusion" about cannabis and people with IDs, I'd simply point to "all of the above", along with every word written and artifact found throughout time. The evidence for Kaneh Botanicals, appears to have been "written in stoned" across the planet, for thousands of years. The healing power of "Natures Pharmaceutical", seems to regulate, relieve and "regenerate" my systems and symptoms, in ways, too many to mention. Having several physical and invisible disabilities, that bring a variety of effects, without the medical benefits cannabis affords me, I would be significantly, more limited. The ability to sleep, the appetite and pain relief are only a small part.

With the clarity and focus it brings, as well as a sense of well-being and peace, I'm better able to tackle some of the extremely difficult and unusual challenges life has handed me, while still maintaining positivity, motivation and sense of humor. I been able to do so many things I never thought I'd be able to do again following my accident and exposure, and things I've never done before. I certainly would never have been able to write any of the contributions that I've been allowed to share here. In the time when I had trouble making coffee, I also could not write a coherent sentence. With the debilitating effects and the fact that there is no currently "approved pharmaceutical interventions to improve" TBI outcomes, the improvement in many areas of my life, has been undeniable.

For anyone who's been on the fence in consideration of turning to Kaneh Bosm as medicine, I leave you with the following statement from the medical experts' findings, found in the National Library of Medicine (NLM):

"The inherent polypharmaceutical properties of cannabis botanicals offer distinct advantages over the current single-target pharmaceutical model and portend to revolutionize neurological treatment into a new reality of effective interventional and even preventative treatment.".

Part 1 - "Is Kaneh-Bosm, Natures Multipurpose Pharmaceutical – Historical, Medical and Scriptural Evidence": https://kolektiva.social/@OutOfExile_IDR_Voice/112934866432502585

Link to sources listed in comments below.

#cannabis #Marijuana #research #TBI #Alzheimers #concussion #PTSD #ADHD #headache #THC #inflammation #pain #MS #Dravet #DravetSyndromes #LennoxGastautSyndrome #LGS

Hola Titánicos, cada 23 de junio se celebra el día mundial del l síndrome de Dravet, también conocido como Epilepsia Mioclónica Severa de la Infancia, con el objetivo de dar visibilidad de lo que supone vivir con esta enfermedad rara.

https://somosdisca.es/el-sindrome-de-dravet-2023/

#salud #disability #discapacidad #sindromededravet #dravet #epilepsia #epilepsiainfantil #niños #enfermedad #enfermedadrara #epilepsiamioclónica #gentetitanica #somosdisca

RT @TueNeuroCampus@twitter.com

Heute ist #TagderSeltenenErkrankungen: Forscherinnen Ulrike Hedrich-Klimosch #HIHTue @uktuebingen@twitter.com, @Nikic_Lab@twitter.com & Friederike Pfeiffer @uni_tue@twitter.com untersuchen #Dravet-Syndrom, eine frühkindliche Epilepsieform @rarediseaseday@twitter.com
PM http://bit.ly/3hnvSwz
Interview http://bit.ly/3tcCPWN

🐦🔗: https://twitter.com/TueNeuroCampus/status/1498248495731519489