Alkeus Pharmaceuticals, Inc.’s Post

Pediatric screening is a simple solution that could prevent premature cardiovascular disease, but we as a healthcare ecosystem are not doing it. Pediatricians are not routinely screening children for FH despite the NHLBI and AAP guidelines. Come be a part of the solution as we discuss this and other topics at this year’s #FamilyHeartSummit24. FamilyHeart.org/Summit

DEBATE CONTINUES! Pediatric Intensivist vs. pediatric hematologist perspective Hyperferritinemic sepsis, MODS (in tropical infections) vs. HLH Critically ill patient >>>Start etoposide alongside steroid (intensivist- big no & hematologist- of course, yes) An unending debate of primary vs. secondary How best to treat HLH in tropical infections ??? This young patient satisfied 7 of 8 HLH criteria. He survived DIC with overt bleed requiring several packets of blood products for several days, raised ICP, and meningo-encephalitis.

Safety of Adalimumab in Pediatric Patients with Polyarticular Juvenile Idiopathic Arthritis, Enthesitis-Related Arthritis, Psoriasis, and Crohn's Disease: A Pediatric Pharmacovigilance Study: https://lnkd.in/e655_dY7 Read more 👉 https://lttr.ai/ARGed #BlogPostAims #ImportantSafetyInformation #MedicalTreatments #PotentialRisks #SideEffects #ComprehensiveUnderstanding #AllergicReactionOccurs #CentralNervousSystem #PeripheralDemyelinatingDiseases #CongestiveHeartFailure

The rare pediatric disease priority review voucher (PRV) program is one of the most important #publichealth policies for patients living with a rare disease. Here’s what you need to know. The PRV program was created in 2012 to spur innovation for rare pediatric diseases. 1 in 2 patients diagnosed with a #raredisease are children - and a third of these children won’t live to see their fifth birthday. We needed a solution to de-risk rare pediatric #drugdevelopment and provide hope for children living with a rare disease - and that’s exactly what the PRV program is designed to do. Over the years, it’s seen enormous success. The program has supported innovations benefiting over 200,000 rare disease patients and helped deliver treatments for 47 unique indications with no existing treatment option.  It’s also enjoyed consistent bipartisan support, having been reauthorized in 2016 and 2020. We’re now approaching its expiration date, which is just over a month away. But despite the program’s widespread and transformative impact, it still has not been reauthorized. That expiration date is September 30. It’s a date that will have huge implications in shaping the future of the rare disease ecosystem. When September 30 passes, we have to ensure that the PRV program passes with it. 

Today ISCT issued a press release celebrating the landmark US FDA approval of RYONCIL, the first MSC therapy approved in the US, for pediatric steroid-refractory acute Graft-versus-Host Disease (GvHD). This milestone marks a breakthrough for the MSC field and a triumph for patients and families battling this severe condition. Read the full release now:https://buff.ly/49M83ZM

#ECG plays a key role in screening for and diagnosing cardiac conditions in children and teens. It's often utilized to investigate potential congenital heart disease and other suspected diseases. But pediatric ECG interpretation presents some unique challenges. #ePeeps #DCARInsights #cardiology

#raredisease #drugdevelopment is hard - and doubly so for #pediatric #rarediseases - those #rare diseases that primarily affect children, and often claim their lives far too early! That is why #incentives like the #rarepediatricdisease #priorityreviewvoucher or #PRV program are so important. We simply *CANNOT let this program expire on September 30!* Too many lives depend on it.

Must read article I just came across: PEDIATRIC HIGH-RISK CONDITIONS Published in EM Clinics of North America [2025; 43:41] CONDITION #1: APPENDICITIS Pearls: -Clinical Decision Rules are often used to make diagnostic and therapeutic decisions -Examples include Pediatric Appendicitis Score [PAS], Alvarado score and Pediatric appendicitis risk calculator [pARC] -Low-risk and high-risk scores generally straight-forward -Issues come up with MID-RANGE scores/risks for appendicitis -How do you manage these children?? More to come re: appendicitis with my daily POSTS Dealing with a pediatric appendicitis case in your INBOX? Text/email me today to have a brief conversation [609-472-0070 / robertbelferpem@gmail.com]

U.S. President Biden signed a bill postponing the sunset date of the rare pediatric disease Priority Review Voucher (PRV) program from September 30, 2024, to December 20, 2024. The bill, the “Continuing Appropriations and Extensions Act, 2025” is a temporary measure to continue the rare pediatric disease PRV program. There are currently other ongoing legislative efforts to renew the program through 2028 and later. We have summarized these proposals here: https://lnkd.in/exn-tcha #PediatricHealth #PriorityReviewVoucher #ContinuingAppropriationsandExtensionsAct2025 Authors: Komal Karnik Nigam, Deborah Cho, and Bryan Walsh.

Get your drug to market 4 months faster with a "Rare Pediatric Disease Priority Review Voucher" but hurry, this program ends September 30, 2024! Refer to FDA’s Draft Guidance on Rare Pediatric Disease Priority Review Vouchers (here: https://lnkd.in/eStfxZSi) for details on what to include in your request. Source (see section 5): https://lnkd.in/ems7aMY3 #RarePediatricDisease #PriorityReviewVoucher #PRV #PediatricDisease #RareDiseases #FDA #Regulations #RegulatoryAffairs