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MIT and UCSF researchers create CRISPR ‘on-off switch’ that controls gene expression without changing DNA dlvr.it/RxNgYv

#Fabrydisease can cause symptoms such as pain and gastrointestinal issues that affect daily life, as well as complications affecting the kidneys, heart, brain and other organs that can lead to long-term challenges. Learn more: bit.ly/3locC36 #1FDSM #RethinkFabry

“Even though many knowledge gaps on the relationship between sleep and CVD [cardiovascular disease] remain to be studied in the coming years, this study provides solid evidence supporting the importance of sleep quality for a better CV health. Read more: ow.ly/5vE250Et2zI

The emotional toll of the pandemic has left many with a sense of stagnation and emptiness. That feeling of muddling through your days, lacking motivation and struggling to focus has a name. nyti.ms/3aAMpKP

Mucopolisaccaridosi, il 15 maggio è la Giornata Mondiale osservatoriomalattierare.it/news/attualita…

Giacomo Chiesi, Head of Global Rare Diseases, discusses how companies in the pharmaceutical industry are positioned to support the global shift towards sustainability in this STAT article: bit.ly/2S3hTm9

Curious about Neuroscience 2021? Here are the answers to some of our most frequently asked questions: bit.ly/3AxiNcD #SfN21

✉️ For our last Hey Kids! Art Crew send an artful message to a friend or family member inspired by artists #OnKawara and #MartinWong. Then share what you made with kids from around the world in virtual sessions this Thursday, August 5. Learn more: mo.ma/3imQWEI

Enrico Piccinini, Head of Region Europe and Emerging Markets, shares a message for the #SickleCellDisease community this #SickleCellAwarenessMonth. #SickleCellMatters #ChiesiGRD

From gene editing to quantum computing, here are seven technologies that are set to shake up science in 2022 go.nature.com/34oAL5u

The EU is a world leader in science, but when it comes to translating science into new treatments, other regions are moving faster. How can we ensure Europe is at the forefront of medical innovation? #WeWontRest

Giacomo Chiesi, Head of Global Rare Diseases, and Gina Cioffi, Senior Manager, Public Affairs, examine findings from our report on the economic burden of #rarediseases and the need for improved policy: bit.ly/396tFF5 PM360 #ChiesiGlobalRareDiseases

For over ten years, Make-A-Wish France has been granting wishes to children, from 3 to 17 years old, suffering from serious illnesses. Granted as a family, the wish brings hope, courage and joy in difficult times.

On Sept. 1, we will host the symposium “What matters most? Improving care in Lysosomal Storage Disorders”, as part of the International Congress of the Society for the Study of Inborn Errors of Metabolism (SSIEM). Check out the program here: ssiem.org

Alpha-mannosidosis is an ultra-rare genetic disease caused by the lack of the enzyme called alphamannosidase (Malm, Nilssen 2019; Borgwardt et al. 2015). Learn more: alphamannosidosis.com (international website) or discoveralphamannosidosis.com (US website)

The Coronavirus has made clear how crucial are #scientificresearch and #pharmaceutical development. That is why we decided to invest on a new #Biotech Center of Excellence and today we had the pleasure of opening the doors of the construction site.

Best Wishes to all!!!

Any day, the Rare Disease day!

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Wonderful project linkedin.com/feed/update/ur…