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Wow !!! A single
#CRISPR Cas9 guide RNA could generate > 7 megabases deletion -> on-target effect (FISH probe) in HEK293 cell line by P53 dependent mechanism on CEP#porphyria disease#genetherapy !!! This is prevented with Cas9 nickase@NatureComms http://nature.com/articles/s4146 …pic.twitter.com/DbQ8lJEJSt
Prikaži ovu nit -
We’re proud to announce a disease education and promotional agreement with
@Alnylam to raise awareness of acute hepatic#porphyria (AHP). Learn more. http://ow.ly/5r1Z50vwkd7 pic.twitter.com/3BvpcImMLn
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CHMP issues positive opinion for
@Alnylam's acute hepatic#porphyria treatment#Givlaari, setting up EU approval in the next couple of monthshttps://bit.ly/2RUB5jD -
Gene silencing as life changing treatment for
#porphyria patients. After 10 years of pain, this lady no longer takes painkillers as the treatment actually reverses the disease!#genesilencing#genomics#innovation#healthnews#givosiran https://www.bbc.co.uk/news/health-47907971 … -
Because I can't go out in the sunlight I'm a hat-wearing, gloves-wearing, little-skin-showing dork in real life.
#porphyria pic.twitter.com/2T8FrsghXi
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FDA approves Givlaari (givosiran) for the treatment of adult patients with acute hepatic
#porphyria http://bit.ly/2KzZgQn pic.twitter.com/L0M6w9P8li
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Doctors have used a new type of medicine called "gene silencing" to reverse a disease that leaves people in crippling pain. Exciting news
#genetics#pharma#porphyria@KingsCollegeLon https://www.bbc.co.uk/news/health-47907971 …pic.twitter.com/E8Dqf85OQi
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I have new pieces of armor. My husband bought me these sun protective gloves with sleeves so I don’t fry as bad when I’m in the car or (god forbid!) outside. We will see how it go’s. I had a coolibar hoodie that I burned through. Cross fingers!
#Porphyria pic.twitter.com/sWqN1jbTAE
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Acute Intermittent
#Porphyria causes recurrent painful abdominal pain crises. Hard to diagnose unless one thinks of it. Thankfully rare but common topic in MRCP exams & more so now with gene-silencing treatment.#MedEd https://www.bbc.com/news/health-47907971 … -
Great to see this article hit top 5 in BBC’s top read!
#porphyria is believed to have played a role in British history with George III and Mary Queen of Scots. https://www.bbc.com/news/health-47907971 … -
APF is pleased to announce that Givlaari (Givosiran) received
#FDA approval for treatment of#acutehepaticporphyria. "This RNAi treatment can prevent intolerable, painful attacks in patients w/ acute#porphyria. It is a game changer." -Kristen Wheeden https://bit.ly/37oGm8M -
September is Pain Awareness Month
@US_Pain. Pain can be silent, but you don’t have to be. For those suffering from acute intermittent#porphyria, severe abdominal pain can last for hours or days. Learn more here: http://bit.ly/2CNwG7q#LetsTalkAboutPain#PAM2019pic.twitter.com/qbLDHqr7lh
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Today Oscar has given a impressive talk about the new advances in the Congenital Eritropoyetic
#porphyria therapy at#ICPP conference, Milan. Joined efford coming from@CICbioGUNE, PTS and@AtlasMolecular.#raredisease https://twitter.com/LabMillet/status/1171419267268431872/photo/1pic.twitter.com/E02drTVuqt pic.twitter.com/UO2qxHP8uT
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PK and PD of the Small Interfering Ribonucleic Acid (
#siRNA), Givosiran, in Patients with Acute Hepatic#Porphyria$ALNY$ARWR$DRNA$ABUS$SLN$AMGN@OliXPharmaceut1#RNAi@RNAi_papers@JMaraganore@Alnylam https://www.ncbi.nlm.nih.gov/pubmed/31994716 pic.twitter.com/X6jFMv339V
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Well done to the
@Alnylam team for securing a positive opinion from the@EMA_News for the treatment of the#raredisease acute hepatic#porphyria which once authorised by the EMA will address unmeet patients needs. https://twitter.com/alnylam/status/1223201101308141570 … -
— التبرع لتكلفة علاج حنون على GoFundMe " أو عن طريق حساب أخوها على بنك مسقط : Account Number: 0378008596230015 (name): Nasser Albusaidi —
#حنون_أقوى_من_البورفيريا ،#حنان_البوسعيدي ،#البورفيريا .@HanoonTweets ,#porphyria#حنون_تبي_مساعدتكم pic.twitter.com/RWeinBlp0NOvo je potencijalno osjetljiv multimedijski sadržaj. Saznajte više -
We’re pleased to announce that we’ve received a positive opinion from the EMA (
@EMA_News) Committee for Medicinal Products for Human Use (#CHMP) for our investigational#RNAi therapeutic for the treatment of the#raredisease acute hepatic#porphyria. -
So depressed, haven't been able to crochet all week after an I.V. gone bad.
#porphyriawarrior#porphyria#porphyriaawareness#chronicillness#chronicpain#chronicillnesswarrior#pain#laheyhospital#ivtherapy https://www.instagram.com/p/B77bhboA2Dp/?igshid=eahhw9c8xx4t … -
I am a Porphyria Warrior ...thank you
@ResidentFOX for bringing awareness to#AcuteIntermittentPorphyria





made me love this show even more!!! #Porphyria https://twitter.com/ResidentFOX/status/1222386267498930176 …
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Wow this episode of
@ResidentFOX@Morris_Chestnut brought life to the disease I was diagnosed in 1995#PORPHYRIA I am a#PorphyriaWarrior




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