[arista]

https://news.sky.com/story/five-mont...ition-12321853


[A five-month-old baby with a spinal disorder
has become the first NHS patient in England
to be treated with a potentially life-saving drug
that can prolong life.

Arthur Morgan was diagnosed with
spinal muscular atrophy (SMA) earlier this month.
He received the one-off US gene therapy,
Zolgensma, at Evelina London Children's Hospital
on 25 May.
The treatment has been called the most
expensive drug in the world - with a list price
of £1.79m per dose.]

[bitontheslide]

all treatments start out expensive

[Ammi]

…it’s incredible and wonderful that something like that can be available on the NHS….I wonder what makes it so expensive, the therapy…

…I hope that the success with him is everything it’s hoped to be …Arthur Morgan……

[bitontheslide]

Originally Posted by Ammi

…it’s incredible and wonderful that something like that can be available on the NHS….I wonder what makes it so expensive, the therapy…

…I hope that the success with him is everything it’s hoped to be …Arthur Morgan……

it's gene therapy, so they would have to formulate a treatment that targets a specific gene

Gene therapy is a promising new technique for treating cancer and genetic disorders that introducing foreign genomic materials into host cells to elicit a therapeutic benefit. The key to success of gene therapy is to create safe and efficient gene delivery vehicles. Besides non-viral vehicles, viral vectors are the most commonly used carriers for delivering DNA because of their high efficiency and safety. Creative Biolabs provides both viral and no-viral vehicles design and construction for basic research and preclinical applications. In addition, we also provide services using gene editing tools to fuel the basic research of gene therapy as well as cell therapy.

[arista]

Originally Posted by bitontheslide

all treatments start out expensive

Yes thats what they are saying
on all Media

[Ammi]

Originally Posted by bitontheslide

it's gene therapy, so they would have to formulate a treatment that targets a specific gene

Gene therapy is a promising new technique for treating cancer and genetic disorders that introducing foreign genomic materials into host cells to elicit a therapeutic benefit. The key to success of gene therapy is to create safe and efficient gene delivery vehicles. Besides non-viral vehicles, viral vectors are the most commonly used carriers for delivering DNA because of their high efficiency and safety. Creative Biolabs provides both viral and no-viral vehicles design and construction for basic research and preclinical applications. In addition, we also provide services using gene editing tools to fuel the basic research of gene therapy as well as cell therapy.

…it’s such incredible progress and another shout out to our NHS that something like this can be available through the system…that little Arthur Morgan can be given a life chance beyond around two years…just so heart warming and hopeful……

[Cherie]

worth every penny

[thesheriff443]

Not to be mr nasty but there always seems to be a but, it’s great news as long as the money spent on this one injection is not taken from other areas of the nhs, rob peter to pay paul.

[bitontheslide]

different budget sheriff, otherwise we would have endless debates on what should and shouldnt get funding

[thesheriff443]

Originally Posted by bitontheslide

different budget sheriff, otherwise we would have endless debates on what should and shouldnt get funding

Oh right, yes it’s a mine field when it comes to funding.

[Ammi]

…I’d guess also that our NHS system being the first to fast track this therapy and to work out a ‘fair deal’ for taxpayers etc…will invite worldwide attention in the medical world, which has to be a positive as well to the U.K. NHS in that it’s a groundbreaking thing…here’s an article…it’s possibly always going to be an expensive treatment because the condition is so rare…it’s a one off treatment with immense life prolonging and enhancing results, which is quite a rare thing as well…when all of the many separate treatments for serious many medical conditions are all added up as well…the total also would be staggering…I recall talking to medical staff about all of the separate aspects of cancer treatments and how huge those are…



The UK's National Health Service (NHS) has fast-tracked the introduction of Novartis’ (NOVN: VX) Zolgensma (onasemnogene abeparvovec) to make it available for babies and young children with spinal muscular atrophy (SMA).

Zolgensma, which has a reported list price of £1.8 million ($2.5 million) per dose and is labelled the most expensive drug in the world, will be available at a price described as ‘fair to taxpayers’ as part of the confidential deal struck by NHS England.

available"Rather than waiting for the National Institute for Health and Care Excellence (NICE) to publish its final guidance, the NHS has taken the step of making it available immediately, an approach that has apparently been backed by the NICE given the importance of administering the one-off treatment as early as possible.

NHS England chief executive Sir Simon Stevens said: “This deal is a life-changer for youngsters with this cruel disease and for their families.

“SMA is the leading genetic cause of death among babies and young children, which is why NHS England has moved mountains to make this treatment available, while successfully negotiating hard behind the scenes to ensure a price that is fair to taxpayers.

“Although the health service is still under real pressure from COVID-19, and NHS England is also focused on leading the national vaccination rollout, today’s agreement is an important reminder that the NHS is looking after millions of other patients too, for whom real medical advances are now possible.”

As many as 80 babies and young children could potentially benefit from Zolgensma each year. The treatment is given as a single intravenous infusion and contains a replica of the missing gene SMN1.

The NHS is currently identifying centers to provide the full-range services required to administer the therapy safely, with new specialist services treating patients in the coming months.

These services require pediatric intensive care and a wide range of other clinical expertise such as neuromuscular, paediatric respiratory medicine, immunology, infectious diseases, hepatology, renal, cardiology and endocrinology.

SMA drugs coming of age
Sally-Anne Tsangarides, general manager at Novartis Gene Therapies in the UK, said: “We thank the entire SMA community of families and clinicians for their efforts to help NICE and NHS England appreciate the unmet need in SMA. We are delighted for them that Zolgensma will soon become available to the families in England who need it.

“Breathing and feeding independently, and developmental milestone achievements like sitting and standing, which have been seen in infants treated with a one-time dose of Zolgensma in clinical studies, are unprecedented in the natural history of the disease.”

The approval of Zolgensma makes it the second drug now available for youngsters with SMA in the UK, after Biogen’s (Nasdaq: BIIB) Spinraza (nusinersen) became available on the NHS to eligible patients in May 2019. Other treatments are also in development for the rare condition.


https://www.thepharmaletter.com/arti...l-on-zolgensma

[bitontheslide]

the medical advancements that are happening at the moment are truly astounding

[smudgie]

Brilliant news.
You can’t put a price on a life.
The family of little Arthur must be overjoyed at this treatment for him.
The hope for all other families in the same situation must be incredible.
The price of the treatment will eventually get less expensive as it is rolled out more.
Gene therapy sounds like the way to go forward.

[arista]

Sadly a Mother with a Baby Edward
who has is 8month with the same condition,
can not have it.

As it is only for under 6month babies.


Ch5HDnews.

[UserSince2005]

When will gays get to have babies on the NHS though?