ZOLGENSMA used for treating Spinal muscular atrophy

ZOLGENSMA
Collected from: https://www.zolgensma.com/

ZOLGENSMA is Modern treatment for spinal Muscular Atrophy (SMA). In children with spinal muscular atrophy (SMA), the SMN1 gene is missing or not working properly the SMN 1 protein . ZOLGENSMA®(onasemnogene abeparvovec-xioi) replaces the function of the missing or non-working SMN1 gene with a new, working SMN gene. ZOLGENSMA is produced to help the body continuously produce SMN1 protein with a single intravenous infusion. It Doesn’t change the DNA. It act by a vector that describe below the article.

How to works ZOLGENSMA

The ZOLGENSMA medication replaces the function of the SMN1 gene to treat SMA that stops the progression of spinal muscular atrophy, with a single time administration.

Mechanism of ZOLGENSMA

Children with SMA are born with a missing SMN1 Gene or nonworking SMN1 gene. ZOLGENSMA is an intravenous infusion that Produce SMN genes where they’re needed, in a child’s body, the ZOLGENSMA help to keep the muscles working as they should.

Why is targeting the genetic root cause so important for ZOLGENSMA ?

Well, the SMN1 gene delivered order to motor neuron cells to make SMN protein. Without enough the protein, the motor neuron cells cannot work properly. That’s lead muscle week. These motor neuron cells are involve for all types of muscle movement that we depend on regularly for breathing, eating, walking, sitting up, walking, and all gait and singing etc.

If there isn’t enough SMN protein being made, motor neurons stop working that leads to a Child to Autism.

The main element of ZOLGENSMA gene therapy are a gene and a vector(a type of virus called AAV9) . The gene is a new, fully functioning SMN gene that’s just waiting to get to work properly. [The vector virus called AAV9 is not harmful for human body.]
Being produced the ZOLGENSMA, The viral DNA is removed and replaced by the new SMN gene,that is then delivered to the SMA child.

SMN = Survival Motor Neuron

AAV9= Adeno associated virus serotype 9

SMA = Spinal Muscular atrophy

FAQ about ZOLGENSMA

What is ZOLGENSMA?

ZOLGENSMA (Onasemnogene abeparvovec) is modern treatment for spinal muscular atrophy. ZOLGENSMA devloped by Swiss drugmaker Novartis pharmaceutical.

What are the indications of ZOLGENSMA?

ZOLGENSMA used for treating Spinal muscular atrophy (lack of SMN1 gene or non working SMN1 gene).

What is the side effect of ZOLGENSMA?

  1. Anaphylaxis
  2. Elevated Liver Enzymes
  3. Vomiting
  4. Acute Liver failure
  5. Decrese platelet count etc

What are the precaution before and After administration of ZOLGENSMA?

  1. Control Any type infection before and after administration of ZOLGENSMA
  2. Give steroid to avoid anaphylaxis

How many times need to infusion of ZOLGENSMA?

ZOLGENSMA nedds One-time intravenous infusion.

What are the age of taking ZOLGENSMA?

It used before 2 years of age of SMA Child.

How long does Zolgensma last?

It prevent motor function decline for at least 5 years. That allow the achievement and maintenance of developmental milestones in children with spinal muscular atrophy (SMA), according to data from long-term clinical trials.

Can Zolgensma increase life expectancy?

Yes, Zolgensma can increase life expectancy and large improvement of Motor function.

ZOLGENSMA Price

Zolgensma price is about 2.1 million USD. It is the most expensive drugs in the world.

Zolgensma price in Bangladesh is about 22 Core, Zolgensma price in India is about 18 Core.

Zolgensma price why so expensive

Zolgensma have such a expensive price tag because it have very expensive research costs. The cost to make Zolgensma is also extremely high. The manufacturing and development cost required for the production of Zolgensma is usually much higher compared with the traditional drugs in the world.

Source : https://www.zolgensma.com/

Author :

Dr Md Noman Islam Nirob, MBBS, Rangpur Medical College, Rangpur, Bangladesh.

CEO, www.doctorsgang.com
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