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Neil Arya Singh_Auckland NewZealand_DMD_ Anti-Myostatin Adnectin (RG6206) program

Kuldeep Singh made this Official Information request to Ministry of Health

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From: Kuldeep Singh

Dear Ministry of Health,
My name is Kuldeep Singh.

This is in regards to my son Neil Arya Singh.He is 7.5 years old.He has a condition called Duchenne Muscular Dystrophy.It is a rare condition and we only have few kids with this condition in NZ.

Duchenne muscular dystrophy (DMD) is a severe type of muscular dystrophy.The symptom of muscle weakness usually begins around the age of four in boys and worsens quickly.

Typically muscle loss occurs first in the upper legs and pelvis followed by those of the upper arms.This can result in trouble standing up.Most are unable to walk by the age of 12.More details can be found using

https://en.wikipedia.org/wiki/Duchenne_m...

https://www.youtube.com/watch?v=7EHdCQn6...

No cure for DMD is known, and an ongoing medical need has been recognized by regulatory authorities.Treatment is generally aimed at controlling the onset of symptoms to maximize the quality of life which can be measured using specific questionnaires,and include:Corticosteroids such as prednisolone and deflazacort lead to short-term improvements in muscle strength and function up to 2 years.Corticosteroids have also been reported to help prolong walking, though the evidence for this is not robust.

Current research includes exon-skipping, stem cell replacement therapy, analog up-regulation, gene replacement, and supportive care to slow disease progression.

A swiss company,Roche is organising this trial at different sites and we were informed by Starship that our boy is eligible for this trial(url below) in Australia.

https://www.pioneeringhealthcare.com/duc...

The trial was supposed to start early February 2019 and now it is stuck at a point where they are unable to take NZ patients at this stage as the study sponsor does not have a third party vendor assigned for travel arrangements for patients.

Can you please advise if we can do something about this I can see him being affected by the condition gradually and something like this can be life changing for him and other kids in NZ.

For his condition,the time is very critical and it is hard for us to wait for something like this as we would like to do everything that we can do as parents for him.

Thanks & Regards

Kuldeep Singh

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Ministry of Health


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Dear Kuldeep

Please accept our apologies for the delay in responding to your query, and
thank you for your email of 27 March 2019 on DMD.

The Ministry of Health does not have funds available for participation in
clinical trials, however in this trial (according to the website) Roche
will cover the cost of study medication, visits, assessments and
reasonable costs of travel.

You may also ask your district health board, and/or Neil's paediatrician
about this.

I hope you find this information useful.

Kind regards

Ministerial Services (OIA)
[Ministry of Health request email]  

Government Services
Office of the Director-General
Ministry of Health
[1]www.health.govt.nz

From:        Kuldeep Singh <[FOI #9939 email]>
To:        OIA/LGOIMA requests at Ministry of Health <[Ministry of Health request email]>,
Date:        27/03/2019 04:10 p.m.
Subject:        Official Information request - Neil Arya Singh_Auckland
NewZealand_DMD_ Anti-Myostatin Adnectin (RG6206) program

--------------------------------------------------------------------------

Dear Ministry of Health,
My name is Kuldeep Singh.

This is in regards to my son Neil Arya Singh.He is 7.5 years old.He has a
condition called Duchenne Muscular
[2]http://scanmail.trustwave.com/?c=5305&am...
is a rare condition and we only have few kids with this condition in NZ.

Duchenne muscular dystrophy (DMD) is a severe type of muscular
dystrophy.The symptom of muscle weakness usually begins around the age of
four in boys and worsens quickly.

Typically muscle loss occurs first in the upper legs and pelvis followed
by those of the upper arms.This can result in trouble standing up.Most are
unable to walk by the age of 12.More details can be found using

[3]https://scanmail.trustwave.com/?c=5305&a...

[4]https://scanmail.trustwave.com/?c=5305&a...

No cure for DMD is known, and an ongoing medical need has been recognized
by regulatory authorities.Treatment is generally aimed at controlling the
onset of symptoms to maximize the quality of life which can be measured
using specific questionnaires,and include:Corticosteroids such as
prednisolone and deflazacort lead to short-term improvements in muscle
strength and function up to 2 years.Corticosteroids have also been
reported to help prolong walking, though the evidence for this is not
robust.

Current research includes exon-skipping, stem cell replacement therapy,
analog up-regulation, gene replacement, and supportive care to slow
disease progression.

A swiss company,Roche is organising this trial at different sites and we
were informed by Starship that our boy is eligible for this trial(url
below) in Australia.

[5]https://scanmail.trustwave.com/?c=5305&a...

The trial was supposed to start early February 2019 and now it is stuck at
a point where they are unable to take NZ patients at this stage as the
study sponsor does not have a third party vendor assigned for travel
arrangements for patients.

Can you please advise if we can do something about this I can see him
being affected by the condition gradually and something like this can be
life changing for him and other kids in NZ.

For his condition,the time is very critical and it is hard for us to wait
for something like this as we would like to do everything that we can do
as parents for him.

Thanks & Regards

Kuldeep Singh

-------------------------------------------------------------------

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