International Niemann–Pick Disease Alliance
ALACHUA, FL — (Marketwired) – 1/17/17 — CTD Holdings, Inc. (OTCQB: CTDH), a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of disease, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track...
The Wylder Nation Foundation, which seeks treatment for acid sphingomyelinase deficiency (ASMD) NPD type A, has partnered with biotech company Perlara: Perlara PBC, a company with a focus on ‘finding treatments for rare diseases previously believed to be too...
Perlara PBC Announces Patient Advocacy Group Partnerships – News Press Release | PharmiWeb.com SAN FRANCISCO, Jan. 10, 2017 /PRNewswire/ — Perlara PBC, a global patient portal and drug discovery platform company for the 1 in 10 families affected by...
Just last week we happily reported that three year old Isadora, had became the first patient of her age to receive enzyme therapy for ASMD Niemann-Pick disease type B. This tremendous news filled everyone connected to the INPDA with a sense...
Update #12 on the AIDNPC clinical programme (arimoclomol in treatment of Niemann-Pick disease type C) (Available for download in English, French, German, Italian, Polish, Spanish) Conference call with patient organizations (22 DECEMBER ’16) SUMMARY 002 Study: Recruitment into the...
A constant smile and strong personality are just two traits of Isadora, a Brazilian girl who has became the first three year old in the world to receive enzyme therapy for Niemann-Pick type B disease. Upon hearing this news...
Marketwired Dec 21, 2016 8:00 AM ALACHUA, FL–(Marketwired – December 21, 2016) – CTD Holdings, Inc.(CTDH), a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of disease, today announced its filing of a Clinical Trial Application (CTA) with...
Pivotal Phase 2/3 Trial of Olipudase Alfa for Adult Patients with Acid Sphingomyelinase Deficiency (Niemann-Pick Disease Type B) to Begin in the UK Oxford, UK – 3 November, 2016 – Sanofi Genzyme, the specialty care global business unit of...
There is so much going on as of late; with various clinical trials beginning and/or expanding, new companies joining the fight against Niemann-Pick disease (Perlara and Novartis), and the partnership of Quinn Madeline and Baby Genes in order to create the first diagnostic carrier...
Clinical Trial Update: Orphazyme expands NPC study to three sites in the USA ( Download Here) Orphazyme’s clinical programme AIDNPC, investigating the orally administered small molecule arimoclomol as a treatment for Niemann-Pick disease type C, is close to opening...