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A very expensive remedy against a strange deadly disease will be made in the country and will be cheaper

2024-02-23T11:44:22.381Z

Highlights: First national medication was launched to combat it, and another will soon be added. The treatment must be covered by social and prepaid works, but there are obstacles. Pain in the hands, feet and abdomen, little or no sweating and skin lesions before the age of 10 are some of the symptoms that outline a strange condition. The disease is of genetic origin characterized by the absence of the enzyme Alpha galactosidase, which helps the digestion of certain fatty substances, causing damage to different organs.


It is for Fabry disease, of genetic origin, which attacks several organs. The first national medication was launched to combat it, and another will soon be added. The treatment must be covered by social and prepaid works, but there are obstacles.


Pain in the hands, feet and abdomen, little or no sweating and skin lesions

before the age of 10

are some of the symptoms that outline a strange condition that is part of the group of Rare Diseases (EPOF) and that

one of each suffers.

40 thousand people

.

This is Fabry Disease, whose

early

diagnosis and treatment is key.

The drama of the patients, in addition to suffering from the disease, is the difficulty in

accessing the medication

that prevents the disorder from progressing.

But these are

drugs that cost millions

, which patients cannot buy nor do pharmacies sell.

They must be guaranteed by social works, prepaid or the State, and in a context of growing crisis in the health system the obstacles increase.

Clarín

spoke with patients whose access to these remedies suffers

intermittency

due to interruptions in coverage, which increases anguish.

The

consequences of this deficiency for health and life

are high.

According to Paula Rozenfeld, a biochemist at the Faculty of Exact Sciences of the University of La Plata, “around 20 years of age, angiokeratomas

grouped in specific areas

(navel, “swimsuit,” mucous membranes), proteinuria, and fever attacks are observed.

, edema, heat and exercise intolerance, auditory and vestibular manifestations.”

The expert adds, in a Conicet publication, that “with the

progression of the disease

, around the fourth decade of life, the target organs that

can cause death

are affected , thus reducing life expectancy: kidney failure, hypertrophy cardiac ventricular and cerebrovascular accidents.

The disease is

of genetic origin

characterized by the absence of the enzyme Alpha galactosidase, which helps the digestion of certain fatty substances, whose accumulation in cells

causes damage to different organs

.

Medication replaces that function that the body lacks.

If this does not occur,

signs and symptoms

can be observed at the cardiovascular, kidney, nervous system and skin levels, progressively.

A mural by Martín Ron inspired by rare diseases, in Parque Patricios.

Diagnosis requires

multidisciplinary collaboration

between geneticists, nephrologists, neurologists, pediatricians, dermatologists and cardiologists.

“Early detection is essential to begin treatment as soon as possible, whether

intravenous enzyme replacement

or

oral therapy

in patients with susceptible mutations,” explains Juan Manuel Politei, neurologist at the Spine Foundation, dedicated to the treatment and research of EPOF.

Medication up to 30% cheaper

In this difficult context, the novelty is that the first Fabry Disease

drug

manufactured in Argentina has just been put on the market , the cost of which is - reportedly

- 30 percent cheaper

than the available imported and most used option.

“The medication is

administered orally

every other day.

The latter differentiates it from enzyme replacement treatments, which involve invasive

intravenous

procedures with a biweekly frequency and reduces the cost for the health system.

In turn, it prevents the formation of antibodies that limit long-term effectiveness,” adds Politei.

Scientists during research into one of the national drugs being launched.

Photo: Pablo Añeli

According to a study published in the New England Journal of Medicine, the drug showed the effectiveness of the treatment by confirming that an early start is associated with

stability of heart and kidney function

.

Other studies showed a good response on

gastrointestinal

and

peripheral nervous system

manifestations .

The drug, called

migalastat

, had already been launched internationally and now the Gador laboratory will produce it in the country.

This medication marks a before and after due to the fact that it is the first to be manufactured in the country, but

it will not be the only one

.

Another treatment developed by the Biosidus laboratory is expected to hit the market in 2025, after completing the clinical trial that went through phase 3 last year and will be developed in the mAbxience laboratory.

Thus, Argentina will become the second country, after Japan, to design and produce

agalsidase beta

, a biosimilar drug - biologically based and at a lower cost - for this pathology.

Now we will have to see what the doctors' position is regarding the

reception

of these new national alternatives, based on the fact that all patients in treatment have been receiving

imported drugs

until now .

Those who will surely not hesitate to assume these launches as good news are the financiers.

P.S.

Source: clarin

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