Ambit Inc.’s Post

🌟 Breaking News in Medical Science! 🌟 Exciting developments from Denali Therapeutics. as they unveil groundbreaking results in the fight against mucopolysaccharidoses (MPS)! 🧬✨ 1️⃣ MPS II & Tividenofusp Alfa (DNL310) Update: After 104 weeks of treatment, patients show remarkable improvement and stabilization, marking a significant milestone in MPS II (Hunter Syndrome) therapy. 🚀💪 The treatment has normalized CSF heparan sulfate and achieved a robust, sustained reduction in neurofilament light chain (NfL), indicating a leap forward in neurodegenerative disease management. 🧠🔬 2️⃣ Launch of DNL126 (ETV:SGSH) for MPS IIIA: Denali kicks off dosing in the Phase 1/2 study of DNL126, targeting MPS IIIA (Sanfilippo Syndrome Type A). Early mouse model studies show promising reductions in heparan sulfate, correlating with cognitive function improvements. 🐁💡 🔍 Why It Matters: Innovation at its Best: Denali’s BBB-crossing enzyme replacement therapies offer new hope, tackling both brain and physical symptoms of MPS diseases. 🌉💊 Community and Science Unite: Collaborative efforts aim for swift approval, bringing new treatment options closer to reality for MPS families. 🤝❤️ 🌈 Looking Ahead: With continued global collaboration and innovative research, Denali is on the fast track to revolutionizing treatment options for MPS diseases, aiming to enhance quality of life for affected individuals and their families. 🌍🙌 Stay tuned for more updates in this transformative journey! 📢 #MPSII #MPSIIIA #TividenofuspAlfa #DNL126 #DenaliTherapeutics #NeurodegenerativeDiseases #InnovativeTherapies #HopeForMPS #ScienceNews #MedicalAdvancements 🌟🔬🧬

Today, Rare Disease Day 2024, represents a global effort towards awareness and equity in social opportunity, healthcare, and access to diagnosis and therapies for people living with a rare disease.   🧬 Rare diseases are not so rare: an estimated 1 in 20 people have a rare disease, and 2 out of 10 people know someone who has one. Accordingly, rare diseases have emerged as a global public health priority and have been at the forefront of biomedical research in recent years.   🩺 Ipsos supports the development and monitors the evolution of treatments in rare diseases – and we thank the rare disease community for their participation in our research programs!   #rarediseaseday #raredisease #biotech #pharmaceutical #digitalhealth #IpsosRareDiseaseCOE

On this year’s Rare Disease Day, we want to acknowledge all of those affected by, or working on treatments for, a rare disease.   Working with some of the world's leading scientific and medical minds, we are dedicated in our mission to develop the first treatment for #TUBB4 #leukodystrophy – a rare, incurable and deadly #disease. We want to thank everyone who has supported SynaptixBio Ltd. helping us to get this far, but days like this only make us want to double our efforts. We are changing lives - not a responsibility we take lightly!   #biotech #pharma #healthcare #lifesciences #rarediseases #drugdiscovery #RareDiseaseDay #RareDiseaseDay #RareDiseaseDay2024 

Today, Rare Disease Day 2024, represents a global effort towards awareness and equity in social opportunity, healthcare, and access to diagnosis and therapies for people living with a rare disease.   🧬 Rare diseases are not so rare: an estimated 1 in 20 people have a rare disease, and 2 out of 10 people know someone who has one. Accordingly, rare diseases have emerged as a global public health priority and have been at the forefront of biomedical research in recent years.   🩺 Ipsos supports the development and monitors the evolution of treatments in rare diseases – and we thank the rare disease community for their participation in our research programs!   #rarediseaseday #raredisease #biotech #pharmaceutical #digitalhealth #IpsosRareDiseaseCOE

Today, Rare Disease Day 2024, represents a global effort towards awareness and equity in social opportunity, healthcare, and access to diagnosis and therapies for people living with a rare disease. 🧬 Rare diseases are not so rare: an estimated one in 20 people have a rare disease, and two out of 10 people know someone who has one. Accordingly, rare diseases have emerged as a global public health priority and have been at the forefront of biomedical research in recent years. 🩺 Ipsos supports the development and monitors the evolution of treatments in rare diseases – and we thank the rare disease community for their participation in our research programs! #rarediseaseday #raredisease #biotech #pharmaceutical #digitalhealth #IpsosRareDiseaseCOE

Today, Rare Disease Day 2024, represents a global effort towards awareness and equity in social opportunity, healthcare, and access to diagnosis and therapies for people living with a rare disease.   🧬 Rare diseases are not so rare: an estimated 1 in 20 people have a rare disease, and 2 out of 10 people know someone who has one. Accordingly, rare diseases have emerged as a global public health priority and have been at the forefront of biomedical research in recent years.   🩺 Ipsos supports the development and monitors the evolution of treatments in rare diseases – and we thank the rare disease community for their participation in our research programs!   #rarediseaseday #raredisease #biotech #pharmaceutical #digitalhealth #IpsosRareDiseaseCOE

Today, Rare Disease Day 2024, represents a global effort towards awareness and equity in social opportunity, healthcare, and access to diagnosis and therapies for people living with a rare disease. 🧬 Rare diseases are not so rare: an estimated one in 20 people have a rare disease, and two out of 10 people know someone who has one. Accordingly, rare diseases have emerged as a global public health priority and have been at the forefront of biomedical research in recent years. 🩺 Ipsos supports the development and monitors the evolution of treatments in rare diseases – and we thank the rare disease community for their participation in our research programs! #rarediseaseday #raredisease #biotech #pharmaceutical #digitalhealth #IpsosRareDiseaseCOE

Today, Rare Disease Day 2024, represents a global effort towards awareness and equity in social opportunity, healthcare, and access to diagnosis and therapies for people living with a rare disease.   🧬 Rare diseases are not so rare: an estimated 1 in 20 people have a rare disease, and 2 out of 10 people know someone who has one. Accordingly, rare diseases have emerged as a global public health priority and have been at the forefront of biomedical research in recent years.   🩺 Ipsos supports the development and monitors the evolution of treatments in rare diseases – and we thank the rare disease community for their participation in our research programs!   #rarediseaseday #raredisease #biotech #pharmaceutical #digitalhealth #IpsosRareDiseaseCOE

Today, Rare Disease Day 2024, represents a global effort towards awareness and equity in social opportunity, healthcare, and access to diagnosis and therapies for people living with a rare disease.   🧬 Rare diseases are not so rare: an estimated 1 in 20 people have a rare disease, and 2 out of 10 people know someone who has one. Accordingly, rare diseases have emerged as a global public health priority and have been at the forefront of biomedical research in recent years.   🩺 Ipsos supports the development and monitors the evolution of treatments in rare diseases – and we thank the rare disease community for their participation in our research programs!   #rarediseaseday #raredisease #biotech #pharmaceutical #digitalhealth #IpsosRareDiseaseCOE

Today, Rare Disease Day 2024, represents a global effort towards awareness and equity in social opportunity, healthcare, and access to diagnosis and therapies for people living with a rare disease.   🧬 Rare diseases are not so rare: an estimated 1 in 20 people have a rare disease, and 2 out of 10 people know someone who has one. Accordingly, rare diseases have emerged as a global public health priority and have been at the forefront of biomedical research in recent years.   🩺 Ipsos supports the development and monitors the evolution of treatments in rare diseases – and we thank the rare disease community for their participation in our research programs!   #rarediseaseday #raredisease #biotech #pharmaceutical #digitalhealth #IpsosRareDiseaseCOE