Ataxia

Human medicines European public assessment report (EPAR): Skyclarys, Omaveloxolone, Status: Opinion

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월요일, 12월 18, 2023
Medicine, Vomiting, Applicant (sketch), ATC, ALT, European Commission, Back pain, Ageing, International, INN, Headache, Friedreich's ataxia, Spasm, Fatigue, EMA, Civil service commission, Patient, Nuclear, Medication package insert, Ataxia, Appetite, CHMP, FA, Marketing, Oxidative stress, Committee, Medical Subject Headings, Diarrhea, Nausea, HIV disease progression rates, Instrumentation, Vaccine, Pharmaceutical industry, Medicinal plants, NFE2L2

The active substance of Skyclarys is omaveloxolone (ATC code: not yet assigned).

Key Points: 
  • The active substance of Skyclarys is omaveloxolone (ATC code: not yet assigned).
  • Omaveloxolone activates the Nuclear factor (erythroid-derived 2)-like 2 (Nrf2) pathway which is involved in the cellular response to oxidative stress.
  • The full indication is:
    Skyclarys is indicated for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older.
  • Detailed recommendations for the use of this product will be described in the summary of product characteristics (SmPC), which will be published in the European public assessment report (EPAR) and made available in all official European Union languages after the marketing authorisation has been granted by the European Commission.

Holiday Pet Dangers

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수요일, 11월 15, 2023
CNS, Xanthine, Holiday, Vomiting, Poisoning, Pet, Urine, Blood pressure, Cat, Toxicity, THC, Toxicology, Coma, Risk, Tremor, Arrhythmia, Chocolate, Hypoglycemia, Xylitol, Metabolic acidosis, Ataxia, New Year's Eve, Milk, Heart rate, Animal, Acute liver failure, Oxycodone, Ethanol, Hypoventilation, Friends, Alaskan Malamute, Caffeine, Seizure, Golden, Kidney failure, Diarrhea, Survival, Death, Tachycardia, Happiness, Paracetamol, Classic, Family, Nightmare, Mouth, Sodium chloride, Central nervous system, Failure, Common, Dog, Hypothermia, Lethargy, Radar, Theobromine, Fireworks, Liquor, Tourism, Makita

MINNEAPOLIS, Nov. 15, 2023 /PRNewswire/ -- The holidays are best spent with family and friends, not taking a trip to the animal hospital. With the trifecta of Thanksgiving, Christmas and New Years Eve coming up, the toxicology experts at Pet Poison Helpline want to warn pet lovers about the many dangers that face pets during this festive season.

Key Points: 
  • With the trifecta of Thanksgiving, Christmas and New Years Eve coming up, the toxicology experts at Pet Poison Helpline want to warn pet lovers about the many dangers that face pets during this festive season.
  • Meinke also contacted Pet Poison Helpline, who then worked with the medical team at BluePearl to identify the lily, diagnose and treat his pets.
  • "Many holiday treats include chocolate, and are often left sitting out on counters and dinner tables," said Dr. Schmid.
  • This year, we want pet lovers to remember the holidays for all the joy and happiness, not an emergency room visit."

The PolG Foundation awards over $3.5M in academic grants to pioneer POLG research

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수요일, 11월 15, 2023
Harvard University, Mitochondrial disease, Natural history, Brain, Research, DNA, MIT, RNA-Seq, Disease, Clinical trial, Patient, Ataxia, Science, Drug discovery, Finnish, Press release, Biomarker, Parent, Participation, Coronavirus Scientific Advisory Board, Mutation, Pharmaceutical industry, Vaccine, Medical imaging, Boston, POLG

Frederik de Nassau, a POLG warrior and son of founding parents, designs a MITO clothing line to raise awareness and funds for The PolG Foundation.

Key Points: 
  • Frederik de Nassau, a POLG warrior and son of founding parents, designs a MITO clothing line to raise awareness and funds for The PolG Foundation.
  • View Shop - https://shopmito.polgfoundation.org/
    A central pillar of the Foundation is to raise funding and provide grants to world-class investigators of basic science, clinical trial research and development of novel therapies for PolG mitochondrial disorders in both paediatric and adult PolG populations.
  • Today, The PolG Foundation is pleased to announce four awardees granted to our first Call for Research Initiatives proposals.
  • With your help and financial backing we can further our ambitious mission by discovering new therapies and a potential cure for PolG.

The PolG Foundation awards over $3.5M in academic grants to pioneer POLG research

Retrieved on: 
수요일, 11월 15, 2023
Harvard University, Mitochondrial disease, Natural history, Research, DNA, MIT, RNA-Seq, Disease, Clinical trial, Patient, Ataxia, Science, Drug discovery, Finnish, Press release, Biomarker, Parent, Participation, Coronavirus Scientific Advisory Board, Mutation, Pharmaceutical industry, Vaccine, Medical imaging, Boston, POLG

Frederik de Nassau, a POLG warrior and son of founding parents, designs a MITO clothing line to raise awareness and funds for The PolG Foundation.

Key Points: 
  • Frederik de Nassau, a POLG warrior and son of founding parents, designs a MITO clothing line to raise awareness and funds for The PolG Foundation.
  • View Shop - https://shopmito.polgfoundation.org/
    A central pillar of the Foundation is to raise funding and provide grants to world-class investigators of basic science, clinical trial research and development of novel therapies for PolG mitochondrial disorders in both paediatric and adult PolG populations.
  • Today, The PolG Foundation is pleased to announce four awardees granted to our first Call for Research Initiatives proposals.
  • With your help and financial backing we can further our ambitious mission by discovering new therapies and a potential cure for PolG.

Voyager Therapeutics Reports Third Quarter 2023 Financial and Operating Results

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월요일, 11월 6, 2023
Research, TRACER, Ataxia, IND, SOD1, Alexion, ALS, Toxicology, GBA1, Neurocrine, G&A, Telephone, FXN, Conference, Neurocrine Biosciences, Capsid, Pfizer, Growth, Novartis, DSM-IV codes, GLP, Neurology, IND Culver Line, Conference call, Therapy, D, Good laboratory practice, Pharmaceutical industry, Natural gas, Voyager

LEXINGTON, Mass., Nov. 06, 2023 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to breaking through barriers in gene therapy and neurology, today reported third quarter 2023 financial and operating results.

Key Points: 
  • ET today -
    LEXINGTON, Mass., Nov. 06, 2023 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to breaking through barriers in gene therapy and neurology, today reported third quarter 2023 financial and operating results.
  • SOD1 gene therapy program for amyotrophic lateral sclerosis (ALS): Voyager expects to identify a lead development candidate in 2023 to support an IND in mid-2025.
  • Collaboration Revenues: Voyager had collaboration revenue of $4.6 million for the third quarter of 2023, compared to $41.1 million for the same period in 2022.
  • ET to discuss the third quarter 2023 financial and operating results.

Playful Puppy Nearly Dies After Ingesting Cigarette

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수요일, 11월 1, 2023
American Cancer Society, Liver injury, Xylitol, Shih Tzu, Poisoning, Seizure, Heart, Health, Vomiting, Life, Human, Family, Toxicology, Great American Smokeout, Smoke, Pet, CNS, Observation, Antiemetic, Tobacco, Hypoglycemia, Hypotension, Hypertension, Risk, Methocarbamol, Gizmo, Tachypnea, Animal, Lethargy, Tremor, Ataxia, Contra body movement, Agitation, Depression, Anticonvulsant, Nicotine, Tachycardia, Heated tobacco product

MINNEAPOLIS, Nov. 1, 2023 /PRNewswire/ --  Arizona dust storms can be dangerous, but one recent gust carried something deadly into Corinna Lopez's backyard – a half-smoked cigarette and butt. Yes, we know cigarettes can be deadly to humans, but in this case the victim was canine. After her four-pound Shih Tzu puppy named Gizmo ingested part of the cigarette, he lost full control of his body movements, vomited and had a seizure.

Key Points: 
  • After her four-pound Shih Tzu puppy named Gizmo ingested part of the cigarette, he lost full control of his body movements, vomited and had a seizure.
  • "One of the neighbors' discarded cigarettes must have blown into the back yard during a recent dust storm," Lopez said.
  • "We were training Gizmo to go outside to pee, but when I went to bring him in, I noticed a cigarette butt stuck to his fur.
  • We had no idea that a cigarette butt could be so dangerous to pets."

Dr. Huda Zoghbi Reveals New Research Into the Mechanisms Driving Rett Syndrome at the 2023 World Congress of Neurology

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수요일, 10월 18, 2023
Spectrum, Seizure, Neuron, Behavior, Human, Genetics, MD, MECP2, DSM-IV codes, Research, WCN, Dysautonomia, Baylor College of Medicine, Disorder, Rett syndrome, Neurology, Brain, Tremor, Ataxia, MECP2 duplication syndrome, Patient, Pharmaceutical industry, Medical imaging

Dr. Zoghbi's groundbreaking research not only enhances our understanding of Rett syndrome but also reveals its connection to a spectrum of neuropsychiatric phenotypes, from autism to bipolar disorders.

Key Points: 
  • Dr. Zoghbi's groundbreaking research not only enhances our understanding of Rett syndrome but also reveals its connection to a spectrum of neuropsychiatric phenotypes, from autism to bipolar disorders.
  • Her research also pinpointed the specific neurons and circuit abnormalities that mediate the various symptoms of Rett syndrome.
  • Moreover, this breakthrough research has far-reaching implications, not only for Rett syndrome but for a broader spectrum of neuropsychiatric disorders.
  • Visit wcn-neurology.com to learn more about Dr. Zoghbi and all the featured research at this year's WCN.

Dr. Huda Zoghbi Reveals New Research Into the Mechanisms Driving Rett Syndrome at the 2023 World Congress of Neurology

Retrieved on: 
수요일, 10월 18, 2023
Spectrum, Seizure, Neuron, Behavior, Human, Genetics, MD, MECP2, DSM-IV codes, Research, WCN, Dysautonomia, Baylor College of Medicine, Disorder, Rett syndrome, Neurology, Brain, Tremor, Ataxia, MECP2 duplication syndrome, Patient, Pharmaceutical industry, Medical imaging

Dr. Zoghbi's groundbreaking research not only enhances our understanding of Rett syndrome but also reveals its connection to a spectrum of neuropsychiatric phenotypes, from autism to bipolar disorders.

Key Points: 
  • Dr. Zoghbi's groundbreaking research not only enhances our understanding of Rett syndrome but also reveals its connection to a spectrum of neuropsychiatric phenotypes, from autism to bipolar disorders.
  • Her research also pinpointed the specific neurons and circuit abnormalities that mediate the various symptoms of Rett syndrome.
  • Moreover, this breakthrough research has far-reaching implications, not only for Rett syndrome but for a broader spectrum of neuropsychiatric disorders.
  • Visit wcn-neurology.com to learn more about Dr. Zoghbi and all the featured research at this year's WCN.

Missing Dog Found High on Pot Brownies

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수요일, 10월 11, 2023
Poisoning, Vomiting, Tree, Cat, Death, Lethargy, AKC, Xylitol, Pet, Diarrhea, Attention deficit hyperactivity disorder predominantly inattentive, Tremor, Ataxia, Weakness, Marketing, Toxicology, Seizure, Chocolate, Bradycardia, Urinary incontinence, Coffee, Dog, Attention, Hypotension, Stomach, Holiday, Medical imaging, Fireworks, Halloween, Dozer

MINNEAPOLIS, Oct. 11, 2023 /PRNewswire/ -- When your pet goes missing, panic sets in. Did someone take them? Were they hit by a car? Are they safe? For Jake Carrigan of Hutchinson, Minn., his missing dog story is more like an old Cheech and Chong comedy bit, except it could have had a tragic ending.

Key Points: 
  • For Jake Carrigan of Hutchinson, Minn., his missing dog story is more like an old Cheech and Chong comedy bit, except it could have had a tragic ending.
  • "I was at work and my brother texted me to ask if my dog Dozer had gotten out of the house," Carrigan recalled.
  • I had him send me the post, and I drove to where the person had seen the dog, but I couldn't find him."
  • I asked my neighbor who I suspect might indulge in such brownies, and he said they didn't come from his house."

SHAREHOLDER ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Design Therapeutics, Inc. - DSGN

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화요일, 10월 3, 2023
LLP, Thrombus, Vein, Ataxia, Design, Pomerantz, Therapy, News, Private investigator

NEW YORK, Oct. 03, 2023 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of Design Therapeutics, Inc. (“Design” or the “Company”) (NASDAQ: DSGN).

Key Points: 
  • NEW YORK, Oct. 03, 2023 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of Design Therapeutics, Inc. (“Design” or the “Company”) (NASDAQ: DSGN).
  • Such investors are advised to contact Robert S. Willoughby at [email protected] or 888-476-6529, ext.
  • The investigation concerns whether Design and certain of its officers and/or directors have engaged in securities fraud or other unlawful business practices.
  • Founded by the late Abraham L. Pomerantz, known as the dean of the class action bar, Pomerantz pioneered the field of securities class actions.