include vitamin D and value can be inputted

include options for prescribed but not used for each of these questions

should be multi-options i.e choose multiple options

fields present in ventilation treatments should also be present in the same section within the baseline forms

please include additional option 'prescribed but not used' for all questions eg CPAP too

if yes 'study date' and options for with or without ventilation support

please include additional option 'prescribed but not used'

fields present in ventilation assistance should also be present within the same section in the baseline form.

'Has respiratory function test been performed and include options yes, no and unknown' free text for sites to input additional comments

include options for 'with brace' and 'without brace' ad options to include answers for both

clarify whether patient wears TLSO or TLSO with x-ray

what does this mean?please define

also please see feedback from UK sites- "- If you click on new consultations since last visit, there is no way of saying who or what kind of consultation and the outcome (for example, an ophthalmology review, a respiratory review…)"

require a drop down menu of specialties to choose from

please include 'date if known' as well.

should be able to select all options since all can be applicable to a patient at the same time.

please include 'unknown' option beside each question

Feedback from UK sites "If patient has not report any change since last visit the 3rd question ‘change in endurance’and 'maximum mobility/motor function achieved' should have a box to say ‘no change’.

rename to vitamin D supplement and Calcium supplement

SMA specific treatments

please combine PEG below, into G Tube/PEG as both as the same (with options for yes, no or unknown)

for age at loss below for each section above that include a question 'lost milestone? with yes, no or unknown.

if yes then (continue with age at loss question).

include an 'unknown' and 'not applicable', (further feedback to be provided)

please include an unknown option

turn this into a question with a yes, no or unknown options. If yes then complete the following-

relabel to 'pre-symptomatic at date of diagnosis'

add these options within the PT section under each scale

we have requested with biogen to deselect this for UK sites.

30-4-20 MAA nusinsersen NICE MAA requirement please highlight as mandatory for UK sites

30-4-20 MAA nusinsersen NICE MAA requirement please highlight.as mandatory for UK sites

30-4-20 MAA nusinsersen NICE, please highlight as mandatory for UK sites- relabel to NGT and include 2 options NGT (tube fed only) and NGT (tube-fed and oral)

30-4-20 MAA nusinsersen NICE requirement please hightight as mandatory for UK sites.

30-4-20 MAA nusinsersen NICE requirement please hightight as mandatory for UK sites.

30-4-20 MAA nusinsersen NICE requirement please hightight as mandatory for UK sites.

30-4-20 MAA nusinsersen NICE requirement please hightight as mandatory for UK sites.

relabel to gastronomy surgery

relabel to gastronomy tube

MAA nusinersen (30-4-20) please remove free text field and provide options 'yes' 'no' 'discontinued' and free text for 'reason for discontinuation'

below the reason for discontinuation field include the following-

'with which other therapy; then include the following options for sites to tick

• Zolgensma IV
• Zolgensma Intrathecal
• Risdiplam

include free text box below that "medication of note:state"

relabel use to 'was this treatment received on the EAP or the MAA'

Below this field, please include

"Date of spinal surgery" "Spinal surgery with growing rods", age (years and months) "single procedure spinal surgery" fusion , age (years and months) "

as per NICE requirements (see page 8 of SMA medical assessment for layout)

FVC percent predicted. We are unable to do this for babies. The form requires a value, sites have had to put 0 to get it through but realistically not done as unable to. Please include free text field for sites to input reason.

NICE/NHS England requiement

• use; Extended access programme or MAA (UK only) • first dose, • parental perceived benefit, • dose given under, • combination therapy, • with which other therapy Has patient been unable to receive intrathecal injection following spinal surgery (followed by yes/no box and free field text to allow for any comments to be noted) PLEASE UPDATE THIS SECTION WITHIN ISMAC VISIT FORMS TOO

NICE/NHS England requirement-to consider for ISMAC?

• date of spinal surgery, (note: there is only 1 date field therefore only 1 surgery date can be captured. If patient has 2 surgeries e.g. rods followed by spinal fusion, the age at surgery may be used to differentiate the two dates). • spinal surgery with growing rods (PLUS AGE), • spinal fusion (PLUS AGE) • use of TLSO.

NICE/NHS England request-"patient has become unable to receive intrathecal injection following spinal surgery field to be added" also free text field for other comments to be recorded.

NICE/NHS England requiement

• use; Extended access programme or MAA (UK only) • first dose, • parental perceived benefit, • dose given under, • combination therapy, • with which other therapy

NICE/NHS England requirement-

• if compliant and clinically indicated: forced vital capacity sitting or lying and percentage of predicted for patients height / age (Note: this will be recorded in free text box as no field exists). • if compliant and clinically indicated: peak cough velocity and % of predicted • type of ventilation used (i.e. NIV, tracheostomy) • estimation of hours of ventilation • number of chest infections per annum

UK sites have requested a definition of G tube

NICE/NHS England requirement-to consider for ISMAC?

• date of spinal surgery, (note: there is only 1 date field therefore only 1 surgery date can be captured. If patient has 2 surgeries e.g. rods followed by spinal fusion, the age at surgery may be used to differentiate the two dates). • spinal surgery with growing rods (PLUS AGE), • spinal fusion (PLUS AGE) • use of TLSO.

NICE/NHS England request-"patient has become unable to receive intrathecal injection following spinal surgery field to be added" also free text field for other comments to be recorded.

UK sites have stated that this is very confusing, are supplements or lab measures being requested? Therefore please clarify. Suggestion to also include bisphosphates if supplements.

Values for vitamin D and Calcium lab test would be good.

doe this refer to spinraza? need to know length of treatment and how many doses.

UK sites have states that "- Even if you click on “no” for any treatment, it keeps flagging that the reason for stopping has not been entered (when it has not even been initiated) " Is this still the case?

from UK sites- "Values for vitamin D and Calcium lab test would be good. "

from UK sites- "Fracture data specifically (and which bones),", include free text for other fractures if not already noted?

from UK sites- "- It always flags up the FVC/%predicted as non-completed…when for the smallest babies this is an impossible measurement to get – it should not be one of the mandatory data" can there be a free text field here to explain why FVC cannot be done in these scenarios?

again please specify..

what does this mean?

Feedback from UK sites "Measure method should including ‘arm span’ . I had to put ulna length as closet for one patient. "

In some cases there are indeed changes in the motor ability so the answer is yes. The question is then followed by the ‘’maximum motor function achieved’’ but the answer to that question is limited to only four options and sometimes the changes that I come across in the follow up visit is not really included in those four options e.g. sitting or standing with support or even head control improvement in some cases.

please incorporate a free text box to note other changes

this is inaccurate, all should be renamed, normal or abnormal.

please define scooter, allow for free field text to define

please define what is meant by regular year round physio.

since there is a separate physio form would it not be best to remove this section, or simplify it further since the clinicians are completing this form not physios

UK sites have requested that a not known/not recorded field be introduced as its not always known.

UK sites have requested that this be ,defined.

Also note 'surgery' after each word to avoid confusion eg tendon surgery, scoliosis surgery etc

is this creatine? should be corrected

UK sites have requested that this be renamed to 'treatments for SMA patients' to prevent confusion eg salbutamol treatment for SMA patients is administered via tablets or syrup, never by spray. A clinician would know that, however an inputter with non-medical knowledge would not necessarily be aware of this and could mistakenly input dosage for salbultamol spray. Therefore, need to be clear that these are treatments for SMA.

UK sites have requested that this be renamed to total daily dose.

sites have outlined that if the no option is selected, then the reason for stopping field still remains even though this is not relevant to the question.

sites have also requested a spinraza date started field.

UK sites have requested a definition aka parameters for this.

UK sites have requested that further fields should be introduced, pre-term or term. rename gestational age to approximate gestational age.

UK sites have requested that muscle biopsy be included as one of the first symptoms

UK sites have requested that this be renamed 'approximate date of symptoms' onset since it's not always possible to have an exact age,

Under orthoses section; standing frame, positioning devices, other devices is noted as per week. Does it mean hours per week?

require clarification as to whether these mean ever performed, also would be useful to include fields to note the last result.

can we please include an option for 'not performed' and include a field underneath that noting reasons why.