NZPompeNetwork

Group for people with Pompe Disease.

[NZPompeNetwork @PompeNowWhat]

Finally! New Zealand is getting its act together!

Funding Of 2nd Generation Enzyme Replacement Therapy (ERT) Nexviazyme (avalglucosidase alfa) For Treating Pompe Disease https://t.co/re9WixGGYs

[CheckRare @CheckRare]

Priya Kishnani, MD, Professor of Pediatrics at Duke University Medical Center, discusses recent clinical trials in Pompe disease #CheckRare #RareDisease #PompeDisease
https://t.co/MgHl13KxIz

[NZPompeNetwork @PompeNowWhat]

Petition for drafting a new Accessibility for New Zealanders Bill - Sign the Petition! https://t.co/q7bB69okwD via @Change

[Maze Therapeutics @MazeInBiotech]

We're thrilled to share our manuscript "Small-molecule inhibition of GYS1 for the treatment of Pompe disease and other glycogen storage disorders,” featured as the cover story of the latest @ScienceMagazine. Congrats to our team on this exciting research! https://t.co/E1pB5qnlc9

[Rachel Smalley @Rachel_Smalley]

It’s not me, Pharmac. It’s you.

This is a long, raw column from Malcolm Mullholland on the loss of his wife, his own cancer diagnosis, and reveals how Pharmac spoke about him in emails during this time.

Steve Maharey, the Chair of Pharmac, issued a statement last month describing my Privacy Act revelations as an “isolated incident” and a “unique situation”.

This is clearly not the case.

Reform must come to Pharmac soon. New Zealand needs - and deserves - a professional, high-performance, drug procurement agency. (paywalled)

[NZPompeNetwork @PompeNowWhat]

@BRJDoyle @Rachel_Smalley Because NZ refuses to pay for decent drugs, we're also at the bottom of the pile with it comes to availability. Makes sense, if I was in business I'd be sending my product to countries that will actually pay.

[NZPompeNetwork @PompeNowWhat]

I see the new health minister is trotting out the same old lines which every other politician adheres to. It's election year, and you won't be getting my vote again!

[Rachel Smalley @Rachel_Smalley]

In an election year you expect political spin and deflection, but the Health Minister’s claim at the weekend that patients and people advocating for access to modern medicines are ‘on the take’ by pharma is a botched and brutal attempt to muddy the waters and deflect criticism away from Pharmac’s shoddy tactics and poor performance.

Minister Ayesha Verrall, for months, has denied all interview requests regarding Pharmac’s performance. Then, when she agreed to a rare interview and spoke with Jack Tame on Q&A at the weekend, she attempted to sow a seed that patients and advocates like myself are influenced or ‘benefiting’ from pharma.

Ayesha Verrall, you are the Health Minister for goodness sake.

This is the same line that Pharmac attempts to feed into the media when sick patients arrive on the steps of the Beehive to petition or protest – 'they are all on the payroll of pharma' apparently.

There is an irony here - just before Ayesha Verrall became a minister, she resigned from a 4.5 year paid role with Pharmac - she worked under CEO Sarah Fitt and Chair Steve Maharey, two people she now says she has full confidence in.

To this day, neither Fitt nor Maharey have commented on the significant failings in leadership and governance that were highlighted in the Pharmac Review. And, worse, OIA’s have revealed communications advice telling them to “look hurried” as they leave select committees to avoid the waiting media, and to “look coy” if they are questioned about the medicines budget.

How is that for 'transparent government'?

Right now, there are significant failings in the screening, diagnosis, and treatment of cancer. In 25 years, we haven’t funded a bowel cancer drug. Kiwis with MS have to ‘prove’ their health has declined in order to get medicines that would have STOPPED their decline, and I recently reported on a pharma company which has packed up and left New Zealand after trying for 13 years to get a meeting with Pharmac – their drug to treat pancreatic cancer is funded in every other western nation on earth.

Pharmac is broken and no longer fit-for-purpose. It’s apathetic leadership, outdated funding methodology, dismissive attitude to patients and advocacy groups, and contempt for the media now renders it a siloed beast that needs to be dismantled, and brought inside the health system. More money won’t help Pharmac. It needs new leadership and a modern fit-for-purpose operating model.

However, what is most concerning right now is the attitude and commentary of the Health Minister – Labour’s fifth Health Minister in 6 years. Minister Verrall, you made the conscious decision to avoid addressing New Zealand’s position at the bottom of the OECD for access to medicines, and instead attempted to muddy the waters around who’s paying patients who are sick or dying with cancer, rare disorders and chronic disease.

Labour, you came into power in 2017 on the mantra of “Be Kind” - what a sham that was. New Zealanders suffering from cancer and disease have enough on their plate without being accused by their Minister of trying to capitalise on their disease by earning a few bucks off pharma.

[NZPompeNetwork @PompeNowWhat]

@1nt39r1ty @Rachel_Smalley @idourmj https://t.co/AyELWxFoM2

[NZPompeNetwork @PompeNowWhat]

@idourmj @Rachel_Smalley Here's hoping you don't have a "personal crisis". You know, something like dying from a disease that could be treated if you lived in other OECD countries.

[NZPompeNetwork @PompeNowWhat]

These data build on the positive results from our Phase 1 study presented earlier this year, demonstrating good tolerability and MZE001’s ability to inhibit GYS1, the enzyme that controls glycogen production. We look forward to advancing this program... https://t.co/iZtoMe45Q4

[NZPompeNetwork @PompeNowWhat]

https://t.co/XO1hyaSr2I

[NZPompeNetwork @PompeNowWhat]

@warwick512 @AnneCla70780917 Yes, they're both as bad as the other.

[Hopetravels @Hopetravels2]

🇺🇸 NIH awards major grant to UB to boost the accuracy of newborn screening.
#PompeNBS #Rare
#Disability #IOPD #LOPD #NBS #NewbornScreening #GSD2 #Lysosomal #LSD
https://t.co/VrD2lvhGVz

[Hopetravels @Hopetravels2]

The amazing Dr Barry Byrne, Pompe researcher, Doctor and advocate
#PompeDisease #Rare #metabolic #GSD2 #Lysosomal
https://t.co/eKdKWNskaa

[EveryLife Foundation @EveryLifeOrg]

#ICYMI
#NewbornScreening saves lives and gives babies born with #rarediseases the best start in life. If you were unable to join us the @NatPress workshop last week, you can catch up with the transcripts and resources at https://t.co/8dvK5Kn3p6 #CoveringRareDisease #journalism

[CheckRare @CheckRare]

Priya Kishnani, MD, Chief, Division of Medical Genetics and Professor of Pediatrics at the Duke University School of Medicine, discusses the Duke Health Rare Disease Center’s work in Pompe disease. @DukeMedSchool @AlliancePompe @pompeindia @RareDiseases
https://t.co/sv3W9S9w6B

[Pompe Support Network @PompeUK]

Next-generation treatment for Pompe Given NICE approval

Nexviadyme, the new Enzyme Replacement Therapy (ERT) for Pompe, developed by Sanofi, has been recommended by NICE, the National Institute for Health and Care Excellence.

https://t.co/TxtFvfI4FU

[Stephanie Fischer @RarePOV]

Just registered to attend #newbornscreening bootcamp virtually to learn how to be an even better advocate for this critical public health program. Travel stipends are available for those who'd like to attend in-person. #NBSbootcamp2022

[Pompe Support Network @PompeUK]

Ketones and Pompe

New studies may have found a way of providing benefits of a Ketogenic diet, without the side-effects, which could help people living with Pompe Disease:

https://t.co/ITsCT1KWls

[UF Health @UFHealth]

UF Health Shands Hospital is ranked in five adult medical specialties by U.S. News & World Report. Once again, UF Health Shands has the most nationally ranked adult and pediatric specialties of any hospital in Florida. https://t.co/yP42rVacFM