FDA approves Sarepta’s Viltolarsen: a milestone for muscular dystrophy treatment

admin 2023年9月18日

Beginner

health, health - beginner

©NS Pharma

Pre-reading questions:

I will read each question. Then, please answer them.

講師がそれぞれの質問を読むので答えましょう。

Do you know what ‘rare disease’ means?
Do you think rare diseases can be cured easily?

Vocabulary:

I will read the words, meanings, and sample sentences. Then, repeat after me.

単語、意味、例文を読みます。講師に続いて音読しましょう。

patient /PEY-shuhnt /

[noun] – a person who is receiving medical care, or who is cared for by a particular doctor or dentist when necessary
The doctor examined the patient thoroughly and recommended a course of treatment for their condition.

rare /rair/

[adjective] – not common or frequent; very unusual
The pharmaceutical company invested significant resources in researching and developing a treatment for this rare form of cancer.

condition /kuhn-DISH-uhn/

[noun] – any of different types of diseases
The doctor prescribed medication to manage the chronic condition and provided lifestyle recommendations to improve overall health.

decision /dih-SIZH-uhn/

[noun] – a choice that you make about something after thinking about several possibilities
The government’s decision to implement stricter regulations was aimed at reducing environmental pollution.

confirm /kuhn-FURM/

[verb] – to prove or state the truth of something that was previously not completely certain
The laboratory tests confirmed that the patient’s symptoms were caused by a bacterial infection.

Article reading:

Please read the whole article. Then, I will check your pronunciation and intonation.

記事を音読しましょう。講師はあなたの発音とイントネーションを確認します。

The US Food and Drug Administration (FDA) has given faster approval to a new drug called Viltolarsen, developed by Sarepta Therapeutics, for the treatment of a specific type of muscular dystrophy. This is great news for patients and families affected by this rare genetic disease. Viltolarsen is designed to target a form of muscular dystrophy called Duchenne muscular dystrophy (DMD), which primarily affects young boys. It helps slow down the progression of the disease.

The FDA’s accelerated approval pathway allows drugs to be used for serious or life-threatening conditions when there are unmet medical needs. The decision to approve Viltolarsen was based on data from clinical trials that showed an increase in dystrophin production in patients treated with the drug. More studies are still needed to confirm the drug’s clinical benefits, but its accelerated approval means that patients can now have access to a potentially life-changing therapy. The FDA’s decision shows their commitment to speeding up the development and availability of treatments for patients who have limited options. With the approval of Viltolarsen, there is renewed hope within the DMD community.

True or False:

Read the sentences and identify if they are true or false based on the article.

文章を読んで、記事に基づいて正誤を答えましょう。

The FDA has granted faster approval to the drug Viltolarsen.
Viltolarsen is developed by Pfizer.
Viltolarsen is specifically for the treatment of diabetes.
DMD primarily affects young boys.
Viltolarsen helps slow down the progression of DMD.

Fill in the blanks:

Choose the correct word from the table then fill in the blanks.

適切な言葉を選んで空欄を埋めましょう。

patient

rare

condition

decision

confirm

The patient was diagnosed with a chronic ___________ that required ongoing medical care.
After careful consideration, she made the ___________ to pursue a career change.
The laboratory test results ___________ the initial diagnosis of the infectious disease.
The doctor took the time to listen to the ___________ and address all their concerns.
The scientist made a ___________ discovery of a previously unknown species of insect.