Vidya CA

I will WIN. Not immediately BUT DEFINITELY.

My son was diagnosed with Hunter syndrome in 2015.
#EndHunterSydrome

[AVROBIO @avrobio]

Today on #MPS Awareness Day we recognize those impacted by these metabolic disorders, specifically #MPSII, also known as #HunterSyndrome. We’re pleased that our collaborators at @OfficialUoM initiated a Phase 1/2 clinical trial in patients with Hunter syndrome.

[REGENXBIO @REGENXBIO]

Today we announced that we have aligned with the U.S. FDA on a path forward toward potential accelerated approval of NAVSUNLI™ (RGX-121), our investigational one-time gene therapy for MPS II (Hunter syndrome). Learn more: https://t.co/OCifDwF2wz

[REGENXBIO @REGENXBIO]

REGENXBIO reports fourth quarter and full year 2023 financial results and recent operational highlights. Learn more: https://t.co/BtxQLgWe82

[Rare Disease Day @rarediseaseday]

Tomorrow is #RareDiseaseDay! Join the Global Chain of Lights! 🌍

The rare disease community will unite to spread solidarity with monuments, offices, schools, and homes lighting up in the Rare Disease Day colours!

Don't miss out on Feb 29, 7 PM local time!

#LightUpForRare

[MPS Society UK, here for those with rare diseases @MPSSocietyUK]

This #RareDiseaseDay, read about Sam who shares what motivates him to turn his disability into an ability. We also covered important topics such as Taskmaster, Manchester United (his favourite football team), writing theatre plays and aerial acrobatics: https://t.co/wiEr2Qsifj

[National MPS Society @MPSSociety]

Today, @REGENXBIO announced the first MPS II subject was dosed by Dr. Escolar, primary investigator for the Phase I/II clinical trial of RGX-121 for treating MPS II. Learn more about this exciting trial below! https://t.co/uHUfhL8ULp

[Prasanna Shirol @Prasannashirol]

Where is the #Justiceforall why exclusive Policy. Disease do not affect selectively.

[National MPS Society @MPSSociety]

Exciting news from @REGENXBIO! Their RGX-121 clinical trial for the treatment of MPS II (Hunter Syndrome) will be enrolling up to six additional patients, and expand to individuals up to 18 years old.
Press release: https://t.co/abltHURhJR
Learn more: https://t.co/ytn9lDaxn5

[ORDIndia @ORDIndia]

Thank you @Orphanet for spotlight on #ORDI We have just completed satisfactory 10 years in our Vision "A Better Life for people with Rare Diseases"
@Prasannashirol @MoHFW_INDIA @NITIAayog @mansukhmandviya @Swasticharan @ICMRDELHI @GoMadhan @lalithseetharam @sbarde @PMOIndia

[ORDIndia @ORDIndia]

Thank you @CMofKarnataka @CmSiddramaiah4 @DKShivakumar @dineshgrao @S_PrakashPatil for support to #RareDisease #patients Congratulations for being 1st state to allocate budget. But Rare disease & Health affects all people of all caste not selectively @Prasannashirol

[CRISPR News 🧬 @CRISPR_News]

Belgian researchers create a new toolbox that makes genomic engineering possible without the use of #CRISPR, offering a solution to some of its major issues. Game-changer for genome research and industry! 🧬🔬💡#CRISPR https://t.co/INBoF3ibsi

[ORDIndia @ORDIndia]

Thank you @RupsaChak @IndianExpress for this article and bring out the challanges of #RareDisease in #India and about Pompe @Prasannashirol @MoHFW_INDIA

[MPS Society UK, here for those with rare diseases @MPSSocietyUK]

The UK regulatory authorities have approved the first ever trial of a revolutionary gene therapy for young children diagnosed with MPS II Hunter. Visit https://t.co/hETv6VDeeS for more information.

[Genetic Alliance UK @GeneticAll_UK]

A very successful drop-in on Tuesday in collab with @CGTCatapult hosted by @LizTwistMP talking to MPs on the potential of gene therapies for rare conditions. 15 MPs joined us to hear from @HaemoSocUK and @alexTLCsupport about the challenges of getting access to these treatments.

[MPS Society UK, here for those with rare diseases @MPSSocietyUK]

A new Gene Therapy Clinical Trial will be opening soon in Manchester
@MFTnhs, for children who have MPS II, Hunters Syndrome. Please contact Professor Rob Wynn for further information on the trial at [email protected] or [email protected]. 💙

[ORDIndia @ORDIndia]

Thank You Modiji forHistoric National Policy for RareDiseases in India. Much needed Hope to neglected RareDiseases Community. We are awaiting the Policy implementation at ground level, kindly intervene to expedite the treatment as we are losing precious lives every passing day

[MPS Society UK, here for those with rare diseases @MPSSocietyUK]

Today is #MPSday and we’re celebrating our MPS heroes. Without a doubt, our ultimate MPS hero is Christine Lavery, a formidable lady who dedicated her life to improving the knowledge, advocacy support and clinical outlook for patients with MPS. #MyMPSHero is Christine Lavery

[ORDIndia @ORDIndia]

Listen to #DrBhat #DrSanjeeva
Challenge of Managing #RareDiseases During #COVID19 Experience from #CenterofExcellence treating 50+ children with #LSD receiving #ERT in #SouthAsian largest center.
https://t.co/9ZmsFf4p7c
@MoHFW_INDIA @PMOIndia @NITIAayog @ICMRDELHI

[Rajesh Rathod @RajeshR12347381]

@AnjuPat72270338 @Harshasheesh02 @narendramodi @PandaVashist @curesmaindia @PMOIndia @mansukhmandviya We urging to Indian govt @narendramodi @PMOIndia sir only you can make wonders in our kids life plz discuss with
@Roche company and provide medicine at affordable prices @curesmaindia #BattleAgainstSMA

[Cure SMA Foundation of India 🇮🇳 @curesmaindia]

Today's #SMArtWarrior is Renuka from Karnataka. She is a fun-loving, adventurous girl who enjoys a good laugh. Narrating her journey, she highlights the importance of physiotherapy. #SMA was not strong enough to stop her from pursuing what she desires! #BattleAgainstSMA #SMAckSMA