Fever

FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy

Retrieved on: 
Montag, März 18, 2024

Metachromatic leukodystrophy is a debilitating, rare genetic disease affecting the brain and nervous system.

Key Points: 
  • Metachromatic leukodystrophy is a debilitating, rare genetic disease affecting the brain and nervous system.
  • The stem cells are collected from the patient and modified by adding a functional copy of the ARSA gene.
  • In children with MLD, treatment with Lenmeldy significantly reduced the risk of severe motor impairment or death compared with untreated children.
  • Eighty five percent of the children treated had normal language and performance IQ scores, which has not been reported in untreated children.

C3 Industries Hosts Grand Opening and Ribbon Cutting Ceremony at New Lakehurst Dispensary in Ocean County, New Jersey

Retrieved on: 
Mittwoch, März 13, 2024

ANN ARBOR, Mich., March 13, 2024 /PRNewswire/ -- C3 Industries ("C3" or "the Company,") a leading multi-state cannabis company dedicated to crafting premium cannabis experiences for consumers, announced the grand opening of its High Profile Lakehurst dispensary which will take place on Friday, March 15, with celebrations throughout the St. Patrick's Day weekend. The official ribbon cutting ceremony will be Saturday March 16, at 4:20 p.m. and will headline a weekend full of celebrations, featuring product specials, vendor tables from Fernway, Verano and TerrAscend, and food trucks and prizes. In addition to these festivities, visitors can also expect a live performance from the Bruce Springsteen cover band, Asbury Fever, on Saturday from 2 to 4 p.m.

Key Points: 
  • "This grand opening celebration epitomizes our commitment to delivering top-notch products and unforgettable moments.
  • "We are thrilled to welcome C3's High Profile dispensary to our vibrant community here in Lakehurst, New Jersey," said Lakehurst Mayor Harry Robbins.
  • We look forward to supporting C3 as they contribute to the growth and prosperity of Lakehurst."
  • High Profile Lakehurst, located at 145 NJ-70, Lakehurst, NJ 80733 , is C3's first retail location in New Jersey, and its 24th dispensary nationwide.

Gaza update: pressure mounts on Israel’s allies to stop supplying the weapons to prevent genocide

Retrieved on: 
Freitag, April 5, 2024

“If you aim at the driver’s side, you will hit the driver full-on,” Chris Lincoln-Jones told the newspaper.

Key Points: 
  • “If you aim at the driver’s side, you will hit the driver full-on,” Chris Lincoln-Jones told the newspaper.
  • It is also thought probable that they were launched from a Hermes drone, made by Elbit Systems – also an Israeli manufacturer.
  • The letter, which the newspaper reports amounts to a legal opinion, says UK arms sales to Israel breach international law and must stop.
  • One obvious way to do that is to stop selling them weapons.


Now many of the aid agencies operating in Gaza have suspended their activities. As Stavropoulou and Schiffling write here, the difficulty of getting aid to the population threatens to make the famine that is engulfing the Gaza Strip worse than it already is.

Read more:
More than 200 aid workers have been killed in Gaza, making famine more likely

UN resolution

  • We spoke with John Strawson, an expert in Israeli politics at the University of East London, who kindly answered our questions about the politics of the situation, especially the US decision not to use its veto to block the resolution after decades of faithfully supporting successive Israeli governments in the security council.
  • Expert Q&A

    Given that Netanyahu has indicated that Israel will not abide by security council resolution 2728 – and the US has said that it’s a non-binding resolution in any case – what does international law say about the enforceability of US resolutions?

  • There appears no reason why resolution 2728 is not legally binding and, if push comes to shove, the security council could order that UN members “take such action by air, sea, or land forces as may be necessary to maintain or restore international peace and security”.

Starvation behind the ‘Iron Wall’

  • According to the Hamas-run Gaza health ministry the total has now passed 33,000 people.
  • Nnenna Awah, whose PhD research at Sheffield Hallam University is in optimising food supply chains, walks us through the different classifications of food insecurity.
  • Kaplan says it is the embodiment of Israel’s “Iron Wall” ideology, developed even before statehood was declared in 1948.
  • Kaplan says that Jabotinsky’s ideological heirs in Likud (including Netanyahu) have rejected more liberal Israeli compromise positions ever since.

Whooping cough is surging in Australia. Why, and how can we protect ourselves?

Retrieved on: 
Freitag, April 5, 2024

Cases are highest in Queensland and New South Wales, with more than 1,000 recorded in each state.

Key Points: 
  • Cases are highest in Queensland and New South Wales, with more than 1,000 recorded in each state.
  • The last time Queensland recorded more than 1,000 cases in three months was the first quarter of 2013.
  • So what is whooping cough, why are cases rising now, and how can you protect yourself?

It’s most dangerous for babies

  • The initial symptoms of whooping cough resemble other cold and flu-like symptoms.
  • However, as the disease progresses into the second week, the coughing fits become worse and more frequent.
  • After or between bouts of coughing, patients may gasp for air and produce the characteristic “whoop” noise.
  • Immunity from these vaccines wanes over time, so it’s also recommended adults receive a booster, particularly those who may come into frequent contact with babies.

Why are cases rising now?

  • Due to COVID measures such as border closures, social isolation and masks, the number of cases declined dramatically during 2020–23.
  • In Australia, cases have been particularly high during this outbreak in children aged 10–14.

A potential superbug

  • Most vaccines used in Australia and other developed countries stimulate your immune system to recognise and target three to five components of the bacteria.
  • These mutations make the bacteria look slightly different to the one used in the vaccine, helping it better hide from the immune system.
  • But in 2008, a new strain appeared in Australia that no longer produced pertactin, one of the components targeted by the vaccine.
  • This means your immune system, like a detective, has one less clue to recognise the bacteria.
  • Read more:
    Low vaccination and immunity rates mean NZ faces a harsh whooping cough winter – what needs to happen

What next?

  • Greater tracking of whooping cough strains, like we do with COVID, is needed to inform future vaccine design and treatments.
  • Importantly, although the bacteria is evolving, current vaccines are still very effective at preventing serious disease and reducing transmission.


Laurence Don Wai Luu does not work for, consult, own shares in or receive funding from any company or organisation that would benefit from this article, and has disclosed no relevant affiliations beyond their academic appointment.

There are new flu vaccines on offer for 2024. Should I get one? What do I need to know?

Retrieved on: 
Mittwoch, April 3, 2024

Although most cases are relatively mild, flu can cause more severe illness in young children and older people.

Key Points: 
  • Although most cases are relatively mild, flu can cause more severe illness in young children and older people.
  • Influenza virtually disappeared from Australia during the first years of the COVID-19 pandemic when public health restrictions reduced contact between people.
  • Since 2022, it has returned to a seasonal pattern, although the flu season has started and peaked a few months earlier than before 2020.

What goes into a flu vaccine?

  • Influenza strains are constantly changing due to genetic mutation, with the pace of genetic change much higher than for SARS-CoV-2 (the virus that causes COVID).
  • Read more:
    Flu vaccine won't definitely stop you from getting the flu, but it's more important than you think

    All current influenza vaccines in Australia contain four different strains (known as quadrivalent vaccines).

What’s different about new flu vaccines?

  • These include egg-based vaccines (Vaxigrip Tetra, Fluarix Tetra, Afluria Quad, FluQuadri and Influvac Tetra), cell-based vaccines (Flucelvax Quad), adjuvanted vaccines (Fluad Quad) and high-dose vaccines (Fluzone High-Dose Quad).
  • Until recently, the process of manufacturing flu vaccines has remained similar.
  • In some flu vaccines, adjuvants (components that stimulate the immune system) are included with the influenza antigens.
  • For example, an adjuvant is used in the Fluad Quad vaccine, recommended for over 65s.
  • Studies suggest adjuvanted influenza vaccines are slightly better than standard egg-based vaccines without adjuvant in older people.
  • Read more:
    Should I get the flu shot if I'm pregnant?

What should I do this year?

  • Influenza vaccines are also recommended in pregnancy to protect both the mother and the baby for the first months of life.
  • Influenza vaccines are widely available, including at GP clinics and pharmacies, while many workplaces have occupational programs.


In older people, a number of vaccines are now recommended: COVID and influenza, as well as one-off courses of pneumococcal and shingles vaccines. In general, most vaccines can be given in the same visit, but talk to your doctor about which ones you need.

Are there side effects?

  • These are expected and reflect the immune system reacting appropriately to the vaccine, and are mostly mild and short-term.
  • People with egg allergies should discuss this with their doctor, but in general, studies suggest they can safely receive any (including egg-based) influenza vaccines.
  • Serious side effects from the influenza vaccine, such as Guillain-Barré syndrome, a neurological complication, are very rare (one case per million people vaccinated).


Allen Cheng receives funding from the National Health and Medical Research Council and the Australian Department of Health and Aged Care. He is a member of the Australian Technical Advisory Group on Immunisation.

Orphan designation: (1R,2S) 6-bromo-alpha-[2-(dimethylamino)ethyl]-2-methoxy-alpha-(1-naphthyl)-beta-phenyl-3-quinolineethanol bedaquiline Treatment of tuberculosis, 26/08/2005 Withdrawn

Retrieved on: 
Mittwoch, April 3, 2024

Orphan designation: (1R,2S) 6-bromo-alpha-[2-(dimethylamino)ethyl]-2-methoxy-alpha-(1-naphthyl)-beta-phenyl-3-quinolineethanol bedaquiline Treatment of tuberculosis, 26/08/2005 Withdrawn

Key Points: 


Orphan designation: (1R,2S) 6-bromo-alpha-[2-(dimethylamino)ethyl]-2-methoxy-alpha-(1-naphthyl)-beta-phenyl-3-quinolineethanol bedaquiline Treatment of tuberculosis, 26/08/2005 Withdrawn

An apple cider vinegar drink a day? New study shows it might help weight loss

Retrieved on: 
Mittwoch, März 13, 2024

An experimental study, released today, looks into whether apple cider vinegar could be effective for weight loss, reduce blood glucose levels and reduce blood lipids (cholesterol and triglycerides).

Key Points: 
  • An experimental study, released today, looks into whether apple cider vinegar could be effective for weight loss, reduce blood glucose levels and reduce blood lipids (cholesterol and triglycerides).
  • The results suggest it could reduce all three – but it might not be as simple as downing an apple cider vinegar drink a day.

What did they do?

  • A control group consumed an inactive drink (a placebo) made (from lactic acid added to water) to look and taste the same.
  • The study was also double-blinded, which means neither the participants or the scientists involved with collecting the data knew who was in which group.

So, what did they find?

  • After a period of three months apple cider vinegar consumption was linked with significant falls in body weight and body mass index (BMI).
  • On average, those who drank apple cider vinegar during that period lost 6–8kg in weight and reduced their BMI by 2.7–3 points, depending on the dose.
  • The authors also report significant decreases in levels of blood glucose, triglycerides, and cholesterol in the apple cider groups.

Is this good news?

  • Firstly, study participants were aged from 12 to 25, so we can’t say whether the results could apply to everyone.
  • And while the researchers kept records of the participants’ diet and exercise during the study, these were not published in the paper.
  • This could be important and influence results if people who did not lose weight quit due to lack of motivation.


Read more:
Turns out the viral 'Sleepy Girl Mocktail' is backed by science. Should you try it?

Any other concerns?


Apple cider vinegar is acidic and there are concerns it may erode tooth enamel. This can be a problem with any acidic beverages, including fizzy drinks, lemon water and orange juice. To minimise the risk of acid erosion some dentists recommend the following after drinking acidic drinks:

Read more:
Apple cider vinegar: is drinking this popular home remedy bad for your teeth? A dentist explains

Down the hatch?

  • This study provides us with some evidence of a link between apple cider vinegar and weight loss.
  • This would provide more robust evidence that apple cider vinegar could be a useful aid for weight loss.


Evangeline Mantzioris is affiliated with Alliance for Research in Nutrition, Exercise and Activity (ARENA) at the University of South Australia. Evangeline Mantzioris has received funding from the National Health and Medical Research Council, and has been appointed to the National Health and Medical Research Council Dietary Guideline Expert Committee.

Press Release: New Phase 2b results for amlitelimab support potential for best-in-class maintenance of response in atopic dermatitis

Retrieved on: 
Montag, März 11, 2024

The safety profile was consistent with Part 1 of the study with amlitelimab being well-tolerated and no new safety concerns identified.

Key Points: 
  • The safety profile was consistent with Part 1 of the study with amlitelimab being well-tolerated and no new safety concerns identified.
  • Overall rates of treatment-emergent adverse events (TEAEs) were 69.8% for continued amlitelimab treatment, 71.9% for the amlitelimab withdrawal-arm and 66.7% for placebo.
  • TEAEs more commonly observed included headache (11.6% amlitelimab continuation, 3.9% amlitelimab withdrawal, 6.7% placebo), upper respiratory tract infection (9.3% amlitelimab continuation, 5.5% amlitelimab withdrawal, 20% placebo).
  • Amlitelimab is currently under clinical investigation, and its safety and efficacy have not been evaluated by any regulatory authority.

Filtering The Facts: The Urology Care Foundation Spreads Awareness for National Kidney Month

Retrieved on: 
Freitag, März 1, 2024

BALTIMORE, March 01, 2024 (GLOBE NEWSWIRE) -- March is National Kidney Month, and the Urology Care Foundation, the official foundation of the American Urological Association (AUA), wants to help share all the tools and information you need to take charge of your kidney health.

Key Points: 
  • BALTIMORE, March 01, 2024 (GLOBE NEWSWIRE) -- March is National Kidney Month, and the Urology Care Foundation, the official foundation of the American Urological Association (AUA), wants to help share all the tools and information you need to take charge of your kidney health.
  • The kidneys also make hormones that help to keep blood pressure stable, make red blood cells and help keep bones strong.
  • Kidney health deserves equal awareness,” said Dr. Timothy Averch, chair of the Urology Care Foundation’s Kidney and Adrenal Health Committee.
  • For National Kidney Month, the Urology Care Foundation is focusing on information about kidney cancer and kidney stones:
    It is estimated that there will be over 81,000 new kidney cancer cases and over 14,000 cancer deaths in the United States in 2024.

Biogen’s QALSODY® (tofersen), the First Therapy to Treat Rare, Genetic Form of ALS, Received Positive Opinion from CHMP

Retrieved on: 
Freitag, Februar 23, 2024

If authorized by the European Commission (EC), QALSODY will be the first treatment approved in the European Union to target a genetic cause of ALS, also known as motor neuron disease (MND).

Key Points: 
  • If authorized by the European Commission (EC), QALSODY will be the first treatment approved in the European Union to target a genetic cause of ALS, also known as motor neuron disease (MND).
  • “The CHMP’s positive opinion reinforces the impact QALSODY can have in SOD1-ALS and further demonstrates Biogen’s commitment to address the unmet needs of people living with ALS and neuromuscular diseases,” said Priya Singhal, M.D., M.P.H., Head of Development at Biogen.
  • Trends towards improvement in the physical abilities of participants who received QALSODY were seen compared to those who received placebo, as measured by the ALS Functional Ratings Scale-Revised (ALSFRS-R).
  • Serious neurologic events, including myelitis and/or radiculitis; papilledema and elevated intracranial pressure; and aseptic meningitis have also been reported.