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How far would you go to save your child’s life? Haoyang was born in Kunming in southwestern China two years ago with a very rare disease: Menkes Syndrome. The disease is a genetic disorder that impacts how copper – which is crucial for brain and nervous system development – is... -
Digital communities have become support networks for patients of rare diseases, as well as professionals. They boost access to research, encourage advocacy, and aid evidence-gathering When Lucinda Andrews learned that her newborn son was only the 16th person in the world known to be diagnosed with an ultra-rare genetic disorder, she...
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Last month, Jessie Jackson’s friends and family sang her happy birthday a little early. Jessie doesn’t turn 30 until Dec. 12, but for the past five years on the anniversary of her diagnosis, her parents have hosted a fundraiser. They weren’t taking any chances. Jessie inherited a rare genetic condition called GM1...
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Rare disease spotlight
Thank you speaking up. We need more people to open their eyes and call for an end to this injustice
Please Help and RETWEET! @SecAzar @realDonaldTrump @SpeakerPelosi @SenSchumer @SEC_Enforcement @mtmdphd @Biotech2050 @MPG61 @VincentRK @ICCS1 @fransaraco @DrMiguelPerales @rarediseaseday @RareDiseases @CheckOrphan @RareCandace @Mohty_EBMT @SeemaCMS @AndyBiotech @IAmBiotech @AP https://twitter.com/grpetersen1/status/1271846385642545152
100,000 American die of COVID 19 and we shut down the county and spend Trillions to fight it, HOWEVER each year a minimum of 135,000 American die from #RareCancers and next to nothing is done to protect the small biotechs which develop Orphan Drugs! http://myelomasurvival.com/myeloma-blog/manipulation-of-the-stocks-of-micro-cap-biotech-companies-is-killing-cancer-patients
CheckOrphan could not agree more. No one looks at the multi-millions that die each year from a #RareDisease
Myopic medical, political and financial focus always does more damage
100,000 American die of COVID 19 and we shut down the county and spend Trillions to fight it, HOWEVER each year a minimum of 135,000 American die from #RareCancers and next to nothing is done to protect the small biotechs which develop Orphan Drugs! http://myelomasurvival.com/myeloma-blog/manipulation-of-the-stocks-of-micro-cap-biotech-companies-is-killing-cancer-patients
This is simple but the critical step in getting immunocompromised pts on the right care path! @CheckOrphan @autoimmunitymd @SjogrensOrg @CreakyJoints @jeffsparks Who do they call?’: Patient, provider education crucial for managing outpatient COVID-19 https://www.healio.com/news/rheumatology/20211122/who-do-they-call-patient-provider-education-crucial-for-managing-outpatient-covid19?utm_medium=social&utm_source=twitter&utm_campaign=sociallinks