International Niemann–Pick Disease Alliance
(Available for Download in English, German, French, Spanish, Italian, and Polish) Interim Information Bulletin SUMMARY 002 Study: All patients have now started treatment with study medicine Orphazyme to present at Spanish NPC Association on June 24th The...
The 21st Niemann-Pick Sielbstilfegruppe family support meeting took place in Kassel last weekend on Saturday 27th May. This was the 17th year that it has been held in the same location, one ideally positioned in the geographical centre of...
Download Here Opportunity for Patients, Families, and Caregivers to Hear Additional Details on Recently Initiated Phase I and Phase I/II Clinical Trials ALACHUA, FL — (Marketwired — May 16, 2017) — CTD Holdings, Inc. (OTCQB: CTDH), a clinical stage...
ORPHAZYME COMPLETES ENROLMENT OF PATIENTS FOR PHASE 3 CLINICAL TRIAL IN NIEMANN-PICK TYPE C DISEASE Copenhagen, 11 May 2017 Download Here Orphazyme ApS, a Danish biotech company developing novel therapies for the treatment of protein misfolding diseases, today announced...
Update #15 on the AIDNPC clinical programme (arimoclomol in treatment of Niemann-Pick disease type C) Conference call with patient organizations (30 MAR ’17) Available for download in English, French, German, Italian, Polish, and Spanish SUMMARY 002 Study:...
Available for Download ALACHUA, FL – (Marketwired) – March 23, 2017 – CTD Holdings, Inc. (OTCQB: CTDH), a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment of disease, today announced that it has begun recruiting patients...
Update #13 on the AIDNPC clinical programme (arimoclomol in treatment of Niemann-Pick disease type C) (Available for download in English, French, German, Italian, Polish, and Spanish) Conference call with patient organizations (26 JANUARY ’17) SUMMARY 002 Study: Recruitment...
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...