Cerapedics Inc.的动态

Today,?September 7, we acknowledge?World Duchenne Muscular Dystrophy Awareness Day?and extend our deepest gratitude to the Duchenne community and C-Path’s?Duchenne Regulatory Science Consortium?members for their tireless dedication to advancing treatments for DMD.?To combat the many challenges posed by this disease,?D-RSC?has created an integrated database of patient-level clinical data from DMD studies,?and several drug development tools?available to the Duchenne community with permissions. Established by?C-Path?and?Parent Project Muscular Dystrophy, D-RSC, in partnership with CDISC,?has also developed standard terminology and authored the?Duchenne Muscular Dystrophy Therapeutic Area User Guide and?the?Cardiac Imaging Supplement currently out for public review until September 30. ? We are proud to continue this work and are grateful for the community’s ongoing collaboration. For more information, visit?c-path.org/programs/d-rsc. To view the Cardiac Imaging Supplement, visit:?https://lnkd.in/g8ujGTsV ? Ramona Belfiore-Oshan, Pat Furlong ? #CPath #DMDawareness #WDAD2024 #musculardystrophy #raredisease #drugdevelopment #datasharing #globalhealth #collaboration?#CDISC

?? Foundation for the Accreditation of Cellular Therapy (FACT) and Joint Accreditation Committee ISCT-Europe & The EBMT (JACIE) have published a preliminary draft of both: ?? FACT-JACIE International Standards for Hematopoietic Cellular Therapy Product Collection, Processing, and Administration, Ninth Edition?(HCT 9 Standards), and ?? FACT-JACIE International Standards for Immune Effector Cells, Third Edition?(IEC) ?? Read the news article and discover more details ?? https://lnkd.in/dduDrkmf The drafts are now open for public comments which will be accepted until 15?December 2024. ?? As the field of #CellularTherapy is continuously evolving, the goal for each new edition of the Standards is to accurately capture and reflect those changes to remain a relevant and useful resource for programs. The comments, suggestions, and feedback solicited during the public comment period are not only welcome, but vital to ensuring that we achieve this goal. ?? ??

?? Join Dr. Vince Clinical Research for an upcoming webinar on managing renal and hepatic impairment trials. These trials are essential for assessing the impact of organ impairment on #pharmacokinetics (PK), but recruitment challenges make them more complex than standard clinical studies. Learn how tools like population PK (PopPK) and physiologically based PK (#PBPK) modeling, combined with adaptive trial designs, can reduce patient burden and improve trial outcomes. ?? Learn more + register here: https://buff.ly/4gASg2T #ADME #DrugDevelopment #ClinicalResearch #CRO #PatientRecruitment #NASH #NAFLD #TherapeuticAreas #TranslationalResearch #NonalcoholicSteatohepatitis

On August 22nd, Alkermes announced the initiation of the Vibrance-2 study, a phase 2 clinical trial evaluating the safety and efficacy of ALKS 2680 compared to placebo in adults with narcolepsy type 2 (NT2). "We are pleased to initiate Vibrance-2, a phase 2 clinical study for adults with narcolepsy type 2, based on the data from our phase 1, proof-of-concept study in this patient population. ALKS 2680 is the most advanced investigational orexin 2 receptor agonist currently in development for narcolepsy type 2," said Craig Hopkinson, M.D., Chief Medical Officer and Executive Vice President, Research & Development at Alkermes. "Across narcolepsy type 1 and narcolepsy type 2, significant unmet need remains, and we look forward to further characterizing the efficacy and safety profile of ALKS 2680 in the Vibrance studies in both of these important patient populations." Read the full article at: https://lnkd.in/eCZBfH_x #narcolepsy #sleepdisorder #phase2 #clinicaltrial #Alkermes

Today we presented exciting preclinical data on our development candidate MRT-8102, demonstrating that it’s a potent, selective, and durable molecular glue degrader (MGD) of NEK7.? ? The data provide preclinical proof of concept that a NEK7 MGD leads to inhibition of the NLRP3 inflammasome and IL-1 release to reduce the effects of inflammation, supporting the potential to address central and peripheral inflammatory disorders. https://bit.ly/4d18243

Really excited to see this progress in a very classic but complex pathophenotype of MS. Lots to consider, but am very curious about biomarker plans once in MS subjects - could it be MTR/MTSat or maybe myelin water and multi shell diffusion in PRL/NonPRL lesions and what will the remyelinating compounds be combined with? #myelin #multiplesclerosis https://lnkd.in/eZUgFb-w

In this paper the effects of bimagrumab, an antibody that blocks activin type II receptors and stimulates skeletal muscle growth are presented. An interesting future concept together with GLP-1 analogues? https://lnkd.in/dbvaji4H

Bioxodes - Did you know? #DYK? Bioxodes is developing a medicine to decrease mortality rates and facilitate a quicker and improved recovery for patients following a hemorrhagic stroke (bleeding in the brain), an often-deadly disease for which no medicines exist at the moment. The drug – called BIOX-101 - is still being tested: first in laboratories, now in patients in clinical tests. But we already know from preclinical studies that: ?? It prevents blood clotting but does not increase the risk of bleeding, ?? It prevents the damaging neuroinflammation at the site of the stroke, and ?? It reduces neuron mortality. These are promising findings, and Bioxodes hopes to show more positive results from trials with patients, which are now taking place in Belgium

We are thrilled to announce our collaboration with Paradigm Medical Communications to offer the first activity of an insightful CME course: "Psoriasis Prime Time: The Role of TYK Inhibition in Psoriasis." In this edition, our expert faculty will: ?Review the unique mechanism of action for allosteric tyrosine kinase 2 (TYK2) inhibitors compared with Janus kinase inhibitors. ?Highlight the efficacy and safety data for the new TYK2 inhibitor in psoriasis. ?Use a case-based discussion to illustrate how TYK2 inhibition can be incorporated into psoriasis treatment. Join us to stay at the forefront of psoriasis management and enhance your clinical practice. https://lnkd.in/gxVjwXmR

Did you know? #DYK? Bioxodes is developing a medicine to decrease mortality rates and facilitate a quicker and improved recovery for patients following a hemorrhagic stroke (bleeding in the brain), an often-deadly disease for which no medicines exist at the moment. The drug – called BIOX-101 - is still being tested: first in laboratories, now in patients in clinical tests. But we already know from preclinical studies that: ?? It prevents blood clotting but does not increase the risk of bleeding, ?? It prevents the damaging neuroinflammation at the site of the stroke, and ?? It reduces neuron mortality. These are promising findings, and Bioxodes hopes to show more positive results from trials with patients, which are now taking place in Belgium. #strokeawareness #voiceofstroke #hemorrhagicstroke #patientcare