Tag: treatment

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Taysha Gene Therapies Receives Rare Pediatric Disease Designation and Orphan Drug Designation for TSHA-102 as a Treatment for Rett Syndrome

Program leverages novel miRARE platform technology used to control transgene expression on a cellular basis

TSHA-102 anticipated to submit Investigational New Drug application in 2021

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-102, an AAV9-based gene therapy in development for the treatment of Rett syndrome. Taysha anticipates that it will submit an Investigational New Drug (IND) application for TSHA-102 to the FDA in 2021.

Rett syndrome is one of the most common genetic causes of severe intellectual disability worldwide, with a prevalence of over 25,000 cases in the U.S. and European Union (EU). It is an X-linked

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By [email protected]_84

Are Antibody Infusions Safe? Eli Lilly Pauses COVID Treatment Trial

KEY POINTS

  • Eli Lilly did not say what symptoms the ill patient developed
  • President Trump was given an antibody cocktail as part of his COVID treatment
  • AstraZeneca earlier paused it’s vaccine trial but has since restarted it in the U.K.

 

Eli Lilly (LLY) on Tuesday paused its coronavirus antibody trial because of a potential safety concern just one day after Johnson & Johnson (JNJ) paused its COVID-19 vaccine trial because of an unexplained illness.

The New York Times reported emails between Lilly and federal government officials concerned the trial testing the benefits of the antibody therapy on hundreds of hospitalized COVID-19 patients. All study patients also were receiving Gilead’s (GILD) trial remedy Remdesivir.

The Times said no details were provided on the number of volunteers who fell ill or any details about their illnesses.

Last week, Lilly reported just two test subjects had serious infusion reactions.

President Donald Trump received

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By [email protected]_84

U.S. pauses Eli Lilly’s trial of a coronavirus antibody treatment over safety concerns

  • Eli Lilly’s phase-three trial of its ACTIV-3 monoclonal antibody treatment for coronavirus has been paused due to potential safety concerns.
  • The ACTIV-3 trial is designed to test a monoclonal antibody developed by Eli Lilly in combination with remdesivir, an anti-viral with emergency use authorization for the virus.
  • It’s one of several ongoing trials, as part of the National Institute of Health’s “Activ” program, designed to accelerate the development of vaccine treatments in partnership with the pharmaceutical industry.



a group of people standing around a plane: In this May 2020 photo provided by Eli Lilly, researchers prepare mammalian cells to produce possible COVID-19 antibodies for testing in a laboratory in Indianapolis.


© Provided by CNBC
In this May 2020 photo provided by Eli Lilly, researchers prepare mammalian cells to produce possible COVID-19 antibodies for testing in a laboratory in Indianapolis.

Eli Lilly’s late-stage trial of its leading monoclonal antibody treatment for the coronavirus has been paused by U.S. health regulators over potential safety concerns, the company confirmed to CNBC on Tuesday.

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“Safety is of the utmost importance to Lilly. We are aware

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By [email protected]_84

Ionis’ inhaled antisense medicine demonstrates potential as a novel treatment for cystic fibrosis | News

CARLSBAD, Calif., Oct. 13, 2020 /PRNewswire/ — Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in antisense therapeutics, announced today that data from a clinical trial of IONIS-ENAC-2.5Rx demonstrated a significant decrease in the expression of epithelial sodium channel (ENaC) in subjects with cystic fibrosis (CF). The study showed a mean 55.6 percent decrease (p<0.05) in ENaC mRNA expression at the 75 mg dose in the multidose segment of the trial. The study represents the first time an antisense medicine delivered directly to the lung via a nebulizer has shown a significant reduction in ENaC messenger RNA levels. In preclinical studies, ENaC mRNA reductions of 40 percent or more resulted in significant improvement in mouse models of CF lung disease.

IONIS-ENAC-2.5Rx is an investigational antisense medicine designed to reduce the expression of ENaC in the lung. ENaC is believed to be hyperactive in cystic fibrosis, which is caused

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By [email protected]_84

Ionis’ inhaled antisense medicine demonstrates potential as a novel treatment for cystic fibrosis

CARLSBAD, Calif., Oct. 13, 2020 /PRNewswire/ — Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in antisense therapeutics, announced today that data from a clinical trial of IONIS-ENAC-2.5Rx demonstrated a significant decrease in the expression of epithelial sodium channel (ENaC) in subjects with cystic fibrosis (CF). The study showed a mean 55.6 percent decrease (p

IONIS-ENAC-2.5Rx is an investigational antisense medicine designed to reduce the expression of ENaC in the lung. ENaC is believed to be hyperactive in cystic fibrosis, which is caused by mutations in the cystic fibrosis transmembrane regulator gene. Data from the Phase 1 study will be presented via e-poster at the 2020 North American Cystic Fibrosis Conference, which will hold virtual sessions and discussions Oct. 21-23.

Cystic fibrosis is a life-threatening disease affecting approximately 30,000 people in the U.S. and about 70,000 worldwide. Although CF is a multisystem disease, the main cause of

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By [email protected]_84

Takeda Group Begins Manufacturing COVID-19 Plasma Treatment Ahead of Approval | Top News

NEW YORK (Reuters) – The Takeda Pharmaceutical Co-led group that is developing a blood plasma treatment for COVID-19 has started manufacturing while the late-stage trial to determine whether it works is ongoing, Takeda Chief Executive Christophe Weber said on Monday.

The group, known as the CoVIg Plasma Alliance, enrolled its first patient in the Phase III trial on Friday after months of delays. It aims to enroll 500 adult patients from the United States, Mexico and 16 other countries and hope to have results by the end of the year.

“The likelihood it works is very high,” Weber said in an interview. “And that’s why we have launched a campaign in order to accelerate the donation of convalescent plasma to manufacture and produce this product.”

The alliance, which includes CSL Behring, Germany’s Biotest AG and other companies, is testing a hyperimmune globulin therapy, which is derived from blood plasma of

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By [email protected]_84

DNA Dental Comprises the Best Dentist in Dallas, TX, Offering Cosmetic, General, and Restorative Dental Care and Treatment Solutions – Press Release

Dallas, TX – Dallas, TX residents who are in search of 5-star and premium dental care and treatment solutions can now rest assured that the team at DNA Dental brings this quality of services to them at affordable rates. The dental clinic in Dallas, TX boasts a team of experienced and professional dental specialists who are constantly improving on their dental practice to ensure that each patient is given the attention and care they deserve.

Focused on improving the oral health and wellness of the members of the community, DNA Dental has proudly expanded on its dentistry practice to offer a wide range of dental solutions, including the very best children’s dentistry solutions in the area. Children who come into the dental clinic will have access to the very best and experienced pediatric dentists who are committed to creating the best dental care and treatment experiences using the latest dental

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By [email protected]_84

Bill Gates says Trump’s coronavirus treatment won’t work for everyone, shouldn’t be called ‘cure’

Microsoft co-founder and billionaire philanthropist Bill Gates said Sunday that the Regeneron antibody cocktail administered to President Trump to treat a case of COVID-19 shouldn’t be referred to as a “cure.”

“The word ‘cure’ is inappropriate because it doesn’t work for everyone,” Gates told NBC’s “Meet the Press.” “But yes, of all the therapeutics, this is the most promising.”

Although an effective vaccine is an ultimate goal for putting an end to the pandemic, Gates noted that monoclonal antibodies allow for treatment that doesn’t require admission to a high percentage of the population.

“With the monoclonal antibodies, it’s only once somebody tests positive, show symptoms and they’re old enough they’re at risk,” Gates said. “That’s the target for this therapeutic.”

He added that if the monoclonal antibody treatments can be approved for

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By [email protected]_84

Bill Gates on Trump virus treatment: The word ‘cure’ is inappropriate because it won’t work for everyone

Microsoft founder and philanthropist Bill Gates said Sunday that the monoclonal antibodies treatment President TrumpDonald John TrumpNorth Korea unveils large intercontinental ballistic missile at military parade Trump no longer considered a risk to transmit COVID-19, doctor says New ad from Trump campaign features Fauci MORE received for his coronavirus infection is not a “cure,” but is the most promising option thus far.

“The word ‘cure’ is inappropriate because it won’t work for everyone,” Gates said Sunday on NBC’s “Meet the Press.” “But of all the therapeutics, this is the most promising.”

Gates added that the Bill & Melinda Gates Foundation has “been working with companies doing antibodies, we reserved factory capacity back in the spring, and now we’re partnered with Eli Lilly, who with Regeneron, has been the fastest to get these antibodies ready.”

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By [email protected]_84

Penile Rash Treatment

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