SANCTUARY’S COFFEE & CONVERSATION: "Supporting Childhood Rare Disease Research" Myrna Haskell, exec editor, spoke w/ Cindy Parseghian, co-founder of the Ara Parseghian Medical Research Fdn. & Sherry Kelly, Ph.D. youtu.be/iBaUJUKhVCA?si… #raredisease #AraParseghian #NiemannPick

Hear from our leadership team about what motivates us to work towards advancing life-changing medicines for patients and families living with challenging diseases. bit.ly/3OMEDzh $CYTH #NiemannPick #NPC1 #AlzheimersDisease

Interested in learning more about our approach? Check out our scientific publications here: bit.ly/3XGqyJj $CYTH #NiemannPick #NPC

Did you see we presented 2 posters at the SSIEM 2024 Annual Symposium? You can check them out here: bit.ly/4cPiCeu $CYTH #SSIEM2024 #NiemannPick #NPC

#NiemannPick disease type C (#NPC) is an ultra-rare, genetic, progressive and fatal neurological disease that occurs in chidlren and adults. Patients experience disease progression in speech, swallow, cognition, fine motor and mobility. Learn more: bit.ly/4dRucHd

Excited to announce we are attending C-Path's First Annual Global Impact Conference! We are grateful for the opportunity and looking forward to it! bit.ly/3znBXFQ $CYTH #NiemannPick #NPC1 #CPath

We had such a great experience at the SSIEM 2024. Thank you for providing us with the opportunity to present! $CYTH #SSIEM2024 #NiemannPick #NPC1 #LysosomalStorageDisease #InheritedMetabolicDisorder

Zevra presented 4 posters of clinical results demonstrating #arimoclomol's safety and efficacy in #NiemannPick disease type C and a poster highlighting pharmacokinetic modeling of nitrogen scavengers and #OLPRUVA in #UreaCycleDisorders: bit.ly/47j0d8A $ZVRA #SSIEM2024

We value our continued partnership with the #NiemannPick community and ongoing efforts to positively impact the lives of individuals affected by #NPC. We're proud to collaborate with the Firefly Fund and the NPC community to highlight the urgent need for an FDA approved therapy.

Cyclo Therapeutics Presents Encouraging Preliminary Safety Data from Ongoing Pivotal Phase 3 Study and Substudy for the Treatment of Niemann-Pick Disease Type C1 (NPC1). bit.ly/4e8Xjpm $CYTH #NiemannPick #NPC1 #SSIEM2024

$ZVRA Zevra社は #NiemannPick C 型疾患の #arimoclomol に関する新しい臨床データと、 (後に続く) #NPC #UCDs #SSIEM2024

Zevra is pleased to present new clinical data on #arimoclomol for #NiemannPick disease type C and pharmacokinetic modeling of #OLPRUVA for #UreaCycleDisorders at @ssiem2024_porto. More details on the conference: bit.ly/3XsAKGw $ZVRA #NPC #UCDs #SSIEM2024

Zevra is a proud sponsor of the International Network for Fatty Acid Oxidation Research and Management (INFORM) 2024 Symposium September 1-2, in Porto, Portugal. Find more details on the event here: bit.ly/3TbGH8f $ZVRA #NiemannPick #NPC

A debilitating disease with fatal outcomes, #NiemannPick patients are in need of a safe and effective treatment option! $CYTH

Check out our informative scientific deck and take a deep dive into our science. bit.ly/3XGqyJj $CYTH #NiemannPick #NPC

Rafael Holdings and Cyclo Therapeutics Enter Into a Definitive Merger Agreement. bit.ly/3yM9qtu $CYTH #NiemannPick #NPC1

Niemann Pick is a rare and devastating inherited lysosomal storage disorder that can affect both children and adults. Learn how we are working to advance a potential solution for this unmet medical need. bit.ly/44t6U5P $CYTH #NiemannPick #NPC

¡¡YA QUEDA MENOS PARA VOLVER A VERNOS!! Corre y no te quedes sin tu dorsal🏃‍♂️🏃‍♀️🏃🏃‍♀️ 👉INSCRIPCIONES entrapol.is/mEZkE Y SI NO PUEDES VENIR, AYÚDANOS A DIFUNDIR 🩵🫶💙 #NiemannPick #carrerarupestrepuebladevalles #carrerasolidaria #alvaronuestrocampeon