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Much-debated drug from Amylyx for Lou Gehrig’s disease wins FDA approval

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Much-debated drug from Amylyx for Lou Gehrig’s disease wins FDA approval

A much-debated drug for Lou Gehrig’s illness received U.S. approval on Thursday from the Meals and Drug Administration.

The nod represents a long-desired victory for sufferers, although it’s prone to renew questions concerning the scientific rigor behind authorities opinions of experimental medicines, because the Related Press has reported. 

The FDA authorised the drug from Amylyx Prescription drugs primarily based on outcomes from one small, mid-stage research, the AP reported.

FDA PANEL VOTES TO RECOMMEND AMYLYX ALS DRUG

Within the small medical research, sufferers with the debilitating illness appeared to progress extra slowly and survive a number of months longer. 

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Take a look at tubes are seen in entrance of Amylyx emblem on this illustration, July 24, 2022. 
(REUTERS/Dado Ruvic/Illustration)

“This approval supplies one other vital therapy possibility for ALS, a life-threatening illness that at present has no treatment,” Dr. Billy Dunn, FDA’s neurology drug director, mentioned in an announcement.

Usually, the FDA requires two giant research or one research with “very persuasive” survival outcomes for approval.

Third authorised treatment for ALS

The newly authorised drug is named Relyvrio.

It is the third U.S.-approved drugs for the therapy of amyotrophic lateral sclerosis, or ALS — which destroys nerve cells wanted for fundamental features resembling strolling, speaking and swallowing.

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“I believe it demonstrates the FDA’s capacity to be facile and I believe it demonstrates numerous tenacity on the a part of ALS sufferers and advocates,” mentioned Dr. Catherine Lomen-Hoerth, an ALS specialist on the College of California San Francisco, the AP reported.

“The corporate actually tried to do all the pieces doable to get this probably promising drug out to sufferers,” she added. 

Amylyx’s drug is the newest in a sequence of neurological medication which have received FDA approval regardless of questionable effectiveness knowledge, based on the AP. 

The company continues to be going through two authorities probes into its approval of the Alzheimer’s drug Aduhelm final 12 months, which has not but been proven to gradual the illness, the AP famous.

In early Sept., FDA advisers — reversing a earlier choice — voted to advocate approval of the drug. 

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However in a web based memo summarizing its choice, the FDA mentioned “regulatory flexibility” was applicable for approving Relyvrio, “given the intense and life-threatening nature of ALS and the substantial unmet want.”

In early Sept., FDA advisers — reversing a earlier choice — voted to advocate approval of the drug. 

The Peripheral and Central Nervous System Medicine Advisory Committee vote was 7-2 in favor of approval after hours of debate relating to the power and reliability of the corporate’s analyses.

This 2018 photo provided by Amylyx shows the company's co-founders Joshua Cohen, left, and Justin Klee in Cambridge, Mass. 

This 2018 photograph offered by Amylyx exhibits the corporate’s co-founders Joshua Cohen, left, and Justin Klee in Cambridge, Mass. 
(Amylyx through AP)

In March, the panel declined to advocate FDA approval for the treatment, as a result of what the group mentioned was an absence of “considerably persuasive” knowledge.

Lately, nonetheless, the FDA confronted quite a lot of strain from ALS sufferers, advocates and members of Congress, the AP famous. 

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The company obtained over 1,300 written feedback from the ALS neighborhood in assist of the therapy.

The corporate didn’t instantly disclose the worth on Thursday, however mentioned it deliberate to announce the knowledge on a name with traders on Friday morning.

FDA approval is anticipated to compel insurers to cowl Amylyx’s treatment.

Amylyx, primarily based in Cambridge, Mass., didn’t instantly disclose the worth on Thursday, however the firm mentioned it deliberate to announce the knowledge on a name with traders on Friday morning, the AP reported. 

In Canada, the place the drug obtained approval in June, the corporate has proposed a value equating to $165,000.

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AMX0035, because the drug can also be recognized, is reportedly a mix of two current merchandise. One is a dietary complement known as taurursodiol, which could be bought on-line. The opposite is a prescription drug known as sodium phenylbutyrate, used to deal with a uncommon sort of metabolic dysfunction.

The mixture is supposed to decelerate ALS.

ALS is a progressive neurodegenerative illness that impacts nerve cells and the spinal wire. There are two sorts of ALS — sporadic, which is the commonest kind within the U.S.; and familial — which suggests it’s inherited, based on ALS.org. Scientists have but to find out the precise explanation for the illness. 

Julia Musto of Fox Information Digital, in addition to the Related Press and Reuters, contributed to this report.

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Horoscope: What’s in Store for You April 22 — April 28, 2024?

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Horoscope: What’s in Store for You April 22 — April 28, 2024?



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With whooping cough cases on the rise, do you need a booster vaccine?

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With whooping cough cases on the rise, do you need a booster vaccine?

As whooping cough cases are surging globally, some may wonder if it’s necessary to get a booster.

Cases of the childhood respiratory disease also known as pertussis are surging internationally and in parts of the U.S., according to a recent report.

Bordetella pertussis is a type of bacteria that causes a very contagious respiratory infection that spreads from person to person through small respiratory droplets, per the CDC.

NEW YORK HEALTH OFFICIALS WARN OF WHOOPING COUGH OUTBREAK AMONG CHILDREN

“Reports indicate that whooping cough outbreaks are surging across Europe, Asia and parts of the United States, including Northern California, marking the largest uptick since 2012, with cases rising sharply since December,” Maggie Rae, president of the Royal Society of Medicine’s epidemiological and public health section in London, told Fox News Digital.

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Bordetella pertussis is a type of bacteria that causes a contagious respiratory infection that spreads from person to person through small respiratory droplets, per the CDC. (iStock)

Prevalence of cases

In the U.K., there were an estimated 555 cases in January of this year and 913 cases in February — compared to 858 cases for all of 2023, according to the UK Health Security Agency.

Cases in China totaled more than 15,000 this January. That’s 15 times higher than the same time period last year, reports stated.

AMID CHILDHOOD PNEUMONIA OUTBREAKS, INFECTIOUS DISEASES EXPERT REVEALS KEY FACTS ABOUT ‘WHITE LUNG SYNDROME’

“Concerns are mounting in Europe, especially in the Netherlands, where 1,800 cases were reported in the first two weeks of April, leading to four deaths, with declining childhood vaccination rates cited as a possible cause by public health officials,” Rae said.

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“This is a very important public health issue, and I would urge those members of the public who require a vaccine for pertussis to take this up.”

Whooping cough is mostly controlled in the United States, although “breakthrough cases” can occur in people who are fully vaccinated. 

Girl coughing

Cases of the childhood respiratory disease known as whooping cough or pertussis are surging internationally and in parts of the U.S., according to a recent report. (iStock)

Clusters of cases in certain parts of the U.S. are expected for this time of year, according to the Centers for Disease Control and Prevention (CDC).

There have been small “clusters” of cases of whooping cough in the U.S., extending from San Francisco to New York City.

CDC RECOMMENDS ADDITIONAL COVID VACCINE FOR ADULTS 65 AND OVER

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A Catholic high school in San Francisco, California, has reported more than 12 cases since January, according to local reports.

The New York City Department of Health and Mental Hygiene estimated 244 cases from Oct. 1, 2023, to Jan. 31, 2024. 

That’s a 200% increase compared to the same time period in the prior year, a recent health advisory stated.

“This is a very important public health issue.”

Most unvaccinated cases involved infants, while most vaccinated individuals were school-aged children

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A majority of adults had an unknown vaccination history, the advisory noted.

The U.S. typically has approximately 20,000 cases of pertussis per year. Yet as people donned masks and practiced physical distancing during the pandemic, annual cases dropped to 6,124 in 2020 and 2,116 in 2021, according to the CDC.

Symptoms and risk factors

Clusters of cases often occur where there are large groups of young people, such as child care centers and schools.

“The symptoms of pertussis are initially like a cold, with a runny nose, and progress to a cough,” Jennifer Duchon, M.D., hospital epidemiologist and director of antimicrobial stewardship at Mount Sinai Kravis Children’s Hospital in New York, told Fox News Digital.

Patients tend to develop a cough that can become severe — sometimes to the point of vomiting, Duchon said.

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Pertussis test

Health care providers typically test for the disease with a nasal swab. (iStock)

“The characteristic ‘whooping’ sound is a gasp that is made when trying to breathe after a long episode of coughing,” she added.

The cough can linger for weeks after a person catches pertussis.

When outbreaks occur, babies are at a high risk of getting sick and dying from the infection, health officials warn.

AS NEW JERSEY INVESTIGATES MUMPS OUTBREAK, EXPERTS SHARE WHAT TO KNOW ABOUT SYMPTOMS, PROTECTION

“Pertussis is most severe in infants 6 months of age or less, especially in infants who were born preterm or are not immunized,” Duchon said.

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Young infants can have a severe cough that impairs their ability to breathe, and can lead to episodes where they vomit, struggle to breathe or even cease breathing after bouts of coughing.”

Girl coughing at doctor

Patients tend to develop a cough that can become severe — sometimes to the point of vomiting, a doctor said. “The characteristic ‘whooping’ sound is a gasp that is made when trying to breathe after a long episode of coughing.” (iStock)

Babies often won’t make that whooping sound, so a warning sign is when their face turns blue as they struggle to breathe, the CDC noted.

The infection can progress to bacterial pneumonia or a condition called pulmonary hypertension, in which heart function is affected by the disease, Duchon warned.

Treatment and prevention

Health care providers typically test for the disease with a nasal swab.

“If pertussis is caught early, patients can take an antibiotic called azithromycin, but this only helps make the disease less severe and does not cure the disease,” Duchon noted.

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MEASLES VACCINATIONS GIVEN IN ’70S AND ’80S MAY HAVE WORN OFF BY NOW, DOCTOR WARNS

“If someone is exposed to pertussis and is at risk for severe disease or had a lot of contact with the ill person, doctors will sometimes recommend a short course of an antibiotic to act as a ‘prophylaxis’ against the disease.”

Currently, there are two kinds of vaccines for whooping cough available in the U.S., according to the CDC.

“The best way to prevent the disease is to make sure that all family members and health care workers are up-to-date on their vaccinations — not only for pertussis, but also other vaccine-preventable diseases,” Duchon told Fox News Digital.

Child vaccine

“Children should get their primary series of vaccines at 2 months, 4 months and 6 months, and then at 15 months through 18 months, and at 4 years through 6 years,” a doctor advised. (iStock)

The DTaP vaccine protects against diphtheria, tetanus and pertussis.

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The Tdap vaccine protects against tetanus, diphtheria and pertussis.

The DTaP vaccine is for babies, while the Tdap “booster” vaccine is for pre-teens, teenagers and adults, per the CDC.

“Before vaccination became available, the disease used to be a major cause of mortality in young children,” Duchon noted.

Specific vaccine recommendations

Due to the high risk to babies, the CDC recommends that pregnant women receive the Tdap vaccine during the 27th and 36th week of pregnancy, regardless of their prior vaccination status.

This prevents 78% of cases in infants younger than 2 months old and decreases hospitalization by 90% for infants younger than 2 months old who are infected with pertussis, according to the CDC.

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“Everyone in close contact with a very young infant should be vaccinated against pertussis.”

It is recommended that babies get immunized with the DTaP vaccine series, which provides immunity for three separate infectious diseases — diphtheria, tetanus and pertussis.

“Children should get their primary series of vaccines at 2 months, 4 months and 6 months, and then at 15 months through 18 months, and at 4 years through 6 years,” Duchon advised.

Tdap vaccine

The Tdap vaccine protects against tetanus, diphtheria and pertussis. (iStock)

Adolescents should receive the Tdap vaccine at 11 to 12 years old to boost their immunity, the CDC recommends.

In children who receive the full series, 98% have full protection against the infection within a year after the last dose, but the response decreases to 71% after five years, the agency states.

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As pertussis immunity wanes from the original vaccination series in childhood, adults should get regular boosters, Monica Gandhi, M.D., professor of medicine and an infectious disease specialist at UCSF/ San Francisco General Hospital, told Fox News Digital.

“Although the exact frequency of the need for booster vaccination has not been precisely worked out, we recommend a tetanus vaccine every 10 years,” she said.

DtaP vaccine

The DTaP vaccine protects against diphtheria, tetanus and pertussis. (iStock)

As the pertussis vaccine comes formulated with tetanus immunization in the form of the Tdap vaccine, many practitioners recommend a pertussis vaccine every 10 years when the booster for tetanus is provided, according to Gandhi.

Other providers may only recommend routine pertussis boosters in certain circumstances, such as for pregnant women or adults who have never been vaccinated, Duchon added.

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“Everyone in close contact with a very young infant should be vaccinated against pertussis,” she said. 

“We call this strategy ‘cocooning,’ where those around the baby form a protective wall against the disease.”

For more Health articles, visit www.foxnews.com/health.

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'Battle of the Diets' Study Reveals the Healthy + Fastest Way to Lose Weight

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'Battle of the Diets' Study Reveals the Healthy + Fastest Way to Lose Weight



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