Quotient Sciences’ Post

🚀 𝑭𝑫𝑨 𝑭𝒂𝒔𝒕 𝑻𝒓𝒂𝒄𝒌𝒔 𝑱𝒐𝒉𝒏𝒔𝒐𝒏 & 𝑱𝒐𝒉𝒏𝒔𝒐𝒏’𝒔 𝑵𝒊𝒑𝒐𝒄𝒂𝒍𝒊𝒎𝒂𝒃 𝒇𝒐𝒓 𝑭𝒆𝒕𝒂𝒍 𝑵𝒆𝒐𝒏𝒂𝒕𝒂𝒍 𝑨𝒍𝒍𝒐𝒊𝒎𝒎𝒖𝒏𝒆 𝑻𝒉𝒓𝒐𝒎𝒃𝒐𝒄𝒚𝒕𝒐𝒑𝒆𝒏𝒊𝒂 🚀 FNAIT is a rare disease in which the immune system of a pregnant individual attacks platelets in the developing fetus, which can affect clotting and bleeding, causing a significant risk of severe bleeding complications to the fetus or newborn. Currently, there are no approved target therapies to manage FNAIT, making the Fast Track designation for nipocalimab a significant milestone in addressing this unmet medical need. The FDA's Fast Track designation expedites the development and review of drugs intended to treat severe conditions with an unmet medical need. This designation comes after promising preclinical and early clinical data for nipocalimab, demonstrating its potential to effectively lower the risk of FNAIT in pregnant patients. Learn more about this potentially life-saving treatment option for neonates. https://lnkd.in/ejvpbX6k #unmeetneeds #pediatric #rarediesease #FDAFasttrack #Childrenshealth #JohnsonandJohnson

Congress is in discussion of reauthorizing the FDA's Pediatric Rare Disease Priority Review Voucher (PRV) Program. This program has been crucial in accelerating the development and approval of treatments for rare pediatric conditions, enabling life-saving therapies to reach children in need more swiftly. The PRV program incentivizes pharmaceutical companies to prioritize rare diseases, a critical area of healthcare. With its expiration imminent, prompt congressional action is necessary to sustain this vital program. Reauthorizing the PRV program is essential for maintaining progress in the fight against rare pediatric diseases and improving patient outcomes. #HealthcareInnovation #PediatricCare #RareDiseases #FDA

great news for Slenyto!

As global leaders with expertise in #raredisease and #pediatric research, we're looking forward to gaining the latest insights on the regulatory and clinical landscape from peers and industry at World Orphan Drug Congress. 👇Comment below or message me if you are interested in meeting with our clinical science leaders during the conference. 👉Learn about our endpoint quality solutions for rare disease #clinicaltrials: https://bit.ly/43Ps7Ys 

As global leaders with expertise in #raredisease and #pediatric research, we're looking forward to gaining the latest insights on the regulatory and clinical landscape from peers and industry at World Orphan Drug Congress. 👇Comment below or message me if you are interested in meeting with our clinical science leaders during the conference. 👉Learn about our endpoint quality solutions for rare disease #clinicaltrials: https://bit.ly/43Ps7Ys 

As global leaders with expertise in #raredisease and #pediatric research, we're looking forward to gaining the latest insights on the regulatory and clinical landscape from peers and industry at World Orphan Drug Congress. 👇Comment below or message me if you are interested in meeting with our clinical science leaders during the conference. 👉Learn about our endpoint quality solutions for rare disease #clinicaltrials: https://bit.ly/43Ps7Ys 

Interesting case report demonstrating the clinical improvements and safety of #deprescribing a strong #anticholinergic drug doxepin: https://buff.ly/3QEUYJA Carina Lundby Barbara Farrell British Pharmacological Society Great use of the ACBcalc online calculator! 💡 https://buff.ly/3QZXiuX #geriatrics #medicine #pharmacy #polypharmacy #elderly

#GCT_NEWS GCT completes enrollment for a Respiratory Pediatric Phase IV Clinical Trial GCT is thrilled to announce the successful completion of enrollment for a Respiratory Pediatric Phase IV Clinical Trial. Notably, we concluded the recruitment phase two months ahead of schedule, a testament to the dedication of our colleagues in Bulgaria and Romania, as well as our motivated Investigators and site staff. This study aims to assess the efficacy and safety of an alcohol-free spray and lozenges in pediatric patients (6-12 years) with sore throat. We are proud to collaborate on this endeavor with our longstanding partner, ZAK-Pharma Dienstleistung Ges.m.b.H. https://lnkd.in/dca7ddj7 #enrollment #patients #pediatric #respiratory #clinicaltrial #CRO #GCT #clinicalresearch #globalclinicaltrials #gctrials #contractresearchorganization #pharma #clinicaloperations #drugdevelopment #GlobalClinicalTrials #clinicaltrials #GCP #biotech #clinicalresearchorganization #collaboration #partnership #sorethroat

In our labs, care isn't just a word, it's the heartbeat of every breakthrough. Witness the fusion where compassion ignites remedies. #galaxypharma #healthtransformation #WellnessJourney #HealthIsHappines #galaxypharma #pharma #medicine #wellbeing #humanbody #infertility #gynecology #oncology

Exciting news from Mirum Pharmaceuticals, Inc. ! 🎉 Livmarli, their groundbreaking treatment for Alagille Syndrome (ALGS), has just received its second FDA approval. This time, it's for treating progressive familial intrahepatic cholestasis (PFIC), which has no other approved therapies! This marks a significant milestone in the field of rare liver disorders. PFIC and ALGS result in liver bile acid buildup, sometimes requiring transplants. PFIC shortens life expectancy, with only half reaching age 10, hence the significance of Mirum's achievement! For a more detailed review of the approval and story behind it, see Fierce Biotech's Article: https://lnkd.in/eXCSP6pC #hepatology #raredisease #pediatrics #biotech #pharmaceuticals #drugapproval #FDA #