Thursday 14th January 2021
Asfotase Alfa COVID-19 PAG Message
At the asfotase alfa (Strensiq) National Authorisation Panel (NAP) meeting in December 2020, the members discussed the impact of COVID-19 on patients’ clinical outcomes (test results) in 2020. Lockdown measures and shielding can lead to a reduction in physical activity, which can in turn affect clinical outcomes, such as weight and ambulation (the ability to walk). Considering the new lockdown measures currently in place in England, we would like to highlight to patients and their carers that shielding is not necessary for Hypophosphatasia (HPP) patients (unless a patient has another medical condition(s) which requires them to shield as detailed in the UK government guidelines). We encourage patients to be active and continue physical activity where it is safe and possible to do so. This advice is supported by the HPP clinical experts on the NAP. If you have any questions or concerns about this guidance, please do not hesitate to contact us.
Tuesday 5th January 2021
Latest information from the BIMDG
The BIMDG have released further answers to some common FAQs about the COVID-19 vaccination programme and how this may affect those living with Inherited Metabolic Disorders. You can read the updated FAQs here.
The UK Government have just announced that England is returning to a national lockdown today. You can see the rules of the current lockdown here.
The Welsh government implemented a national lockdown on Sunday 20th December. You can see the rules that apply to Wales during their lockdown here.
From today, Scotland has entered a new lockdown. You can see the rules that apply to Scotland here.
Northern Ireland entered a national lockdown on Saturday 26th December. You can see the current restrictions in Northern Ireland here.
Friday 11th December 2020
Latest information from the BIMDG
The BIMDG have just released their answers to some common FAQs about the COVID-19 vaccination programme and how this may affect those living with Inherited Metabolic Disorders. You can read them here.
Monday 7th December 2020
Latest information from the BIMDG
The BIMDG have recently released a statement concerning the eligibility of patients living with Inherited Metabolic Disorders for the newly approved vaccine for COVID-19. You can read the statement here.
You can also see the answers to the some of the common FAQs about the vaccine here.
Wednesday 2nd December 2020
Over the Christmas period, England, Wales, Scotland and Northern Ireland have agreed to new rules to allow families across the UK to mix. To see the new rules for the Christmas period, please see here.
From Wednesday 2nd December, England is reverting to a tier based system after the month long national lockdown. You can see the rules of the different tiers here and you can see which tier you live in here. The tier system will be reviewed every two weeks and adjustments may be made to the rules and/or the tier that your area is located in.
From Friday 4th December, the rules in Wales are changing. You can see the updated guidance here.
Between Tuesday 27th December and Wednesday 10th December, a lockdown is in place in Northern Ireland. You can see the restrictions for the circuit breaker here.
After the circuit breaker, new rules will be in place from Friday 11th December and you can see a summary of the new rules here.
Monday 9th November 2020
Latest information from the BIMDG
The latest government guidance on covid19 identifies two groups of people who are more at risk of complications if they develop the infection and has provided advice for these groups:
- Clinically vulnerable people are those who are:
- aged 60 or over (regardless of medical conditions)
- under 60 with an underlying health condition listed below (that is, anyone instructed to get a flu jab each year on medical grounds):
- chronic (long-term) mild to moderate respiratory diseases, such as asthma, chronic obstructive pulmonary disease (COPD), emphysema or bronchitis
- chronic heart disease, such as heart failure
- chronic kidney disease
- chronic liver disease, such as hepatitis
- chronic neurological conditions, such as Parkinson’s disease, motor neurone disease, multiple sclerosis (MS) or cerebral palsy
- a weakened immune system as the result of certain conditions or medicines they are taking (such as steroid tablets)
- being seriously overweight (a body mass index (BMI) of 40 kg/m2 or above)
The advice for clinically vulnerable people is that they:
- should be especially careful to follow the social distancing (and lockdown) rules and minimise their contacts with others
- should continue to wash their hands carefully and more frequently than usual and maintain thorough cleaning of frequently touched areas in their home and/or workspace.
- wear a face covering in indoor settings where social distancing may be difficult, and where they will come into contact with people they do not normally meet.
- Clinically extremely vulnerable people will include those with an Inherited Metabolic Disorder AND one or more of the following issues:
- Treatment with immunosuppression therapies sufficient to significantly increase the risk of infection
- Significant neurological impairment which includes difficulty swallowing, impaired ability to cough or clear the airway
- Decompensated liver disease
- A previous solid organ transplant (e.g. heart, liver, or kidney)
- Significant renal failure – on dialysis or chronic kidney disease (stage 5, eGFR < 15 ml/min)
- Long-term life dependency on ventilation – either via tracheostomy or CPAP/BiPAP
- Severe lung disease (eg. requiring supplemental home oxygen)
- People who have, or are undergoing treatment for, some specific cancers (see full guidance below for details)
- People who have had a bone marrow or stem cell transplant in the last 6 months or who are still taking immunosuppression drugs
- Women who are pregnant with significant heart disease
- Brittle / poorly controlled disease with frequent hospitalisations for management of severe metabolic decompensation
- Other patients with complex disease or multiple co-morbidities deemed clinically extremely vulnerable by their metabolic centre.
Any individual patient who feels they have been put on the clinically extremely vulnerable list in error, or who feels they may have been omitted from this list in error should contact their treating metabolic centre directly for advice.
Key points for individuals who are deemed ‘clinically extremely vulnerable’ and their families / household members:
- Clinically extremely vulnerable people are advised to stay at home as much as possible, except to go outdoors for exercise or to attend essential health appointments.
- We urge people to continue to seek support from the NHS and other health providers for existing health conditions and any new health concerns.
- People in this group may wish to meet up with one other person from outside their household or support bubble, for example, to exercise in an outdoor public place, but it is suggested that they always try to do so as safely as possible.
- People in this group are strongly advised to work from home. If they cannot work from home, they are advised to not attend work for this period of restrictions. People in this situation may be eligible for Statutory Sick Pay (SSP), Employment Support Allowance (ESA) or Universal Credit. Letters are being sent to people who are clinically extremely vulnerable which may act as evidence in accessing this support.
- Most children originally identified as clinically extremely vulnerable no longer need to follow this advice. People should speak to their GP or specialist clinician, if they have not already done so, to understand whether their child should still be classed as clinically extremely vulnerable.
- People in this group should avoid all non-essential travel by private or public transport. This includes not travelling to work, school, the shops or pharmacy. They should ask others to collect and deliver e.g. medicines and shopping – seeking support from friends, family, or a volunteer, including NHS Volunteer Responders.
- These new measures will apply nationally for 4 weeks up to 2 December.
- Other people living in a household with someone who is clinically extremely vulnerable are not advised to follow this guidance.
- NHS Volunteer Responders can help with shopping and medicines delivery, as well as a regular, friendly phone call and transport to and from medical appointments. Call 0808 196 3646 between 8am and 8pm to arrange support or visit NHS Volunteer Responders.
- DHSC are writing letters to everyone currently on the Shielded Patient List (SPL) to make them aware of the new guidance. We expect these to arrive with patients from mid-week this week.
Monday 26th October 2020
In the initial coronavirus wave, extremely vulnerable people were told to shield. However, shielding was discontinued from the beginning of August. For the latest guidance on what to do if you are a vulnerable person or live with someone who is vulnerable, please see the following link: https://www.gov.uk/government/publications/guidance-on-shielding-and-protecting-extremely-vulnerable-persons-from-covid-19/guidance-on-shielding-and-protecting-extremely-vulnerable-persons-from-covid-19
Tuesday 20th October 2020
Wales are implementing a national lockdown between Friday 23rd October 2020 and Monday 9th November 2020 to curb the rise of coronavirus cases in Wales. To see the rules associated with this lockdown, please click on the following link: https://gov.wales/coronavirus-firebreak-frequently-asked-questions
Thursday 15th October 2020
England has now adopted a three tier system to assess the coronavirus risk level in different parts of England. Depending on the risk level of a certain area, different rules apply.
To see the different alert levels and what they mean, please see here: https://www.gov.uk/guidance/local-covid-alert-levels-what-you-need-to-know
If you live in England, you can see the coronavirus risk level of your area and the rules that apply to you here: https://www.gov.uk/find-coronavirus-local-restrictions
In Northern Ireland, a new set of rules have been implemented for four weeks from Friday 16th October to curb the rise of coronavirus cases in Northern Ireland. To see the latest rules, please see the link: https://www.nidirect.gov.uk/articles/coronavirus-covid-19-regulations-guidance-what-restrictions-mean-you
In Wales, a number of local lockdowns have been implemented to help stop the rise of coronavirus cases. To see the list of places in Wales with local lockdowns and their rules, please see the link: https://gov.wales/local-lockdown
On Friday 9th October, Scotland introduced further measures to try and reduce the spread of coronavirus. To see the latest restrictions in Scotland, please see here: https://www.gov.scot/news/new-moves-to-stop-covid-19-spread/
Friday 21st August 2020
Schools in England, Wales and Northern Ireland are due to restart in the coming weeks, with schools in Scotland having all returned, which may be nerve-wracking for many families and children.
For full guidance on schools reopening and the measures that the government has asked schools to take, including making provisions for children with special needs or who have been shielding due to their disorder or the condition of a family member, please see the links below.
- Full Guidance: https://www.gov.uk/government/publications/actions-for-schools-during-the-coronavirus-outbreak/guidance-for-full-opening-schools
- Guidance for Pupils with Education, Health and Care Plans (EHC Plans): https://www.gov.uk/government/publications/actions-for-schools-during-the-coronavirus-outbreak/guidance-for-full-opening-schools#B
- Full Guidance: https://gov.wales/back-school-plans-september-coronavirus
- Strategy for Reopening of Schools: https://www.gov.scot/publications/excellent-equity-during-covid-19-pandemic-strategic-framework-reopening-schools-early-learning-childcare-provision-scotland/pages/1/
- Preparing for Schools Reopening: https://www.gov.scot/publications/coronavirus-covid-19-guidance-preparing-start-new-school-term-august-2020/
- Full Guidance: https://www.nidirect.gov.uk/articles/coronavirus-covid-19-advice-schools-colleges-and-universities
For further guidance on plans that your school are implementing in accordance with government guidance, please contact your school directly.
If you would like any help with explaining your disorder to your school, we can provide information about your condition to give to your school. Please contact us on email@example.com for further details on how we can help.
Friday 31st July 2020
On Saturday 1st August, shielding is due to be paused for people across the UK with the exception of some local lockdowns and additional ones which have come into force today. Please note that all clinically extremely vulnerable people should receive a text and/or a letter if their area is going into local lockdown.
For information on current shielding guidelines and lockdowns, please see the links below.
National Guidance for ‘Pausing’ Shielding:
- Those in regional or local lockdowns must follow local guidance.
Local Lockdown Guidance:
North West (including Greater Manchester, East Lancashire and West Yorkshire) Lockdown Guidance:
Leicester, Oadby and Wigston Lockdown Guidance:
- Lockdown is due to be lifted on Monday 3rd August 2020.
- Shielding should continue and will be reviewed on Saturday 1st August 2020 when further guidance is given.
- You should not “pause” shielding in line with national guidance in these areas.
Luton Lockdown Guidance:
- Lockdown is due to be lifted on Saturday 1st August 2020.
- Shielding is currently in place until Monday 17th August 2020.
Monday 8th June
Webinar: Covid-19 and Inherited Metabolic Disorders
Friday 5th June
Update for those who have been advised to shield
The advice has now changed so that people who are shielding can now choose to leave their home if they wish to spend time outdoors.
If you’re shielding, you need to be careful when outdoors and must maintain strict social distancing. This means keeping 2 metres apart (3 steps) from anyone outside your household.
If you’re shielding, time outdoors can be alone or with members of your own household. If you live alone, you may choose to spend time outdoors with one person from another household (keeping 2 metres apart at all times). Ideally, this should be the same person each time.
It’s OK if you want to remain inside your own home because you do not feel comfortable with going into public spaces or with having any form of contact with other people who don’t normally live with you. However, careful time outside in the fresh air can help clear the head and lift your mood.
To read the latest guidance please click here
Monday 25th May
Call for Questions
The next webinar in our coronavirus update series will take place on Thursday 11th June.
As we are starting to see some of the lockdown restrictions lifted, we know you’ll have questions and concerns about the road to recovery. If you have any questions that you’d like our expert panel to address, please email them to firstname.lastname@example.org by 5pm on Friday 29th May.
More details about the webinar, including the time and how to join, will follow soon.
Tuesday 21st April
The recording of our webinar ‘Facing Coronavirus Together: Tapping into the Resilience of the Rare Disease Community’ can be found here.
Monday 20th April
The British Paediatric Neurology Association Update
The British Paediatric Neurology Association (BPNA) has developed a guidance document regarding the management of paediatric neurology patients during the coronavirus pandemic. You can view the advice here: https://bpna.org.uk/_common/show_unpro_doc.php?doc=Covid19andPaediatricNeurology20200417_242ca380def669e6d61d20b0c72eade8.pdf
Thursday 16th April
It is likely that most (but not necessarily all) adult patients with Nephropathic Cystinosis will be in the “highly vulnerable” group. Advice about isolation and shielding changes frequently; and so the government website should be consulted: https://www.gov.uk/coronavirus
Adults may encounter more complications than children. If they get painful and persistently red eyes, they may need to contact their local eye department to see if they need an urgent review. Otherwise, continuation of existing ocular medication is required.
As for children, Cystinosis is not a condition that predisposes to more severe disease. However, as with any infection, if they do become ill, especially if they are unable to tolerate fluids, there is, of course, an increased risk for dehydration and worsening electrolyte abnormalities for which they may need to be seen and may benefit from IV fluids. This is not specific for COVID-19; but true for any infection.
We recommend visiting the following website, which is updated frequently with advice from specialist societies, for further guidance: https://www.kidneycareuk.org/news-and-campaigns/coronavirus-advice/
Your local Nephrology team will advise you about supply of medications including Cystagon and eye drops, which you should continue to take as usual. Home delivery services may have to be activated for those not already using this system.
Thursday 9th April
Webinar: Facing Coronavirus Together: Tapping into the Resilience of the Rare Disease Community.
Our next webinar will take place on Tuesday 21st April, 12.30-1.30pm BST
The link to join the webinar on the day is: https://zoom.us/j/647190348
We are working with Duchenne UK and IMD Clinical Psychologists to share the top 5 tops for helping parents and carers, and the top 5 tips for helping your children through the lockdown. There will be the opportunity to ask any questions about the best way to cope during the current crisis and extended period of isolation.
A recording will be available afterwards for those unable to join us live.
Wednesday 8th April
Newborn Screening and Bloodspot Testing during the Coronavirus Crisis
We have contacted Public Health England and received the following communication in regards of Newborn Screening and bloodspot testing.
Newborn Screening should be continuing as usual. All babies should be receiving their screening test on time (wherever possible), and those that test positive should be referred as per usual pathways. Contingency planning is underway with the laboratories to prevent any delays.
Parents should continue to accept screening, and they should expect any screen-positive babies to receive the usual standard of care that would normally be provided.
Public Health England will be monitoring the situation so, may take different action if notified of any significant issues.
If you have any concerns or would like to raise any issues please contact us
Thursday 2nd April
Statement regarding the impact of coronavirus (covid-19) on the asfotase alfa (Strensiq) Managed Access Agreement
NICE and NHS England and NHS Improvement have taken advice from clinical experts to review the potential impact of COVID-19 on the delivery of the Managed Access Agreement (MAA) asfotase alfa (Strensiq) for treating paediatric-onset Hypophosphatasia (HPP) [HST6]. This statement sets out considerations for patients who are already receiving treatment and those patients who have not yet started treatment. For more information please click here.
Monday 23rd March
The recording of our webinar on Inherited Metabolic Disorders and Coronavirus can be found here.
Wednesday 18th March
Ibuprofen and coronavirus
There is currently no strong evidence that ibuprofen can make coronavirus (COVID-19) worse. But until we have more information, take paracetamol unless your doctor has told you paracetamol is not suitable for you. If you are already taking ibuprofen or another non-steroidal anti-inflammatory (NSAID) on the advice of a doctor, do not stop taking it without checking first.
1) Paracetamol is the first line choice
2) If this is not effective, or you cannot obtain any paracetamol then ibuprofen remains the second line treatment for most people for a high fever (there may be some people with asthma or heart disease who have been specifically advised to avoid it).
3) Remaining well hydrated is important.
4) An adult who is otherwise reasonably well, may not require specific treatment (other than trying to stay comfortable) for a low grade fever.
5) In children (particularly younger children) and patients with Glutaric Acidura Type 1 (GA1) treating the fever would be considered the important thing to do (as the risk of uncontrolled fever would be deemed higher than any smaller potential risk of ibuprofen). If in doubt, contact your metabolic team.
Tuesday 17th March
Statement from Nutricia for IMD patients requiring medical nutrition
We know that many people rely on our products and we take this responsibility very seriously. We would like to reassure you that the availability of our products and the continuity of patient care through our Nutricia Homeward Service is a priority.
We are fulfilling orders as usual and have supplies for everyone who has been prescribed our products. Please continue to order your products in their usual quantities as this will help you, and everyone else, get regular, uninterrupted supplies.
We have been monitoring developments closely since the beginning of the COVID-19 outbreak and working hard to ensure the continuity of our products and services. We are in regular dialogue with our factories and suppliers so that we are in the best possible position to respond quickly to the evolving situation.
We recognise this is an extremely anxious time for everyone so please be assured we are doing everything we can to make sure that you get the products you need.
Thank you for your patience.