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1 Study offers new insights into "Niemann-Pick type C disease"
2 Orphazyme to showcase data on arimoclomol in Niemann-Pick disease Type C during the 2021 Annual WORLDSymposium™
3 Arimoclomol Demonstrates Sustained Effect in Niemann-Pick Disease Type C
4 When Drug Development for Rare Disease Hit Setback, Parents Were Stung
5 „Fat jam" in the cell
6 Review Highlights Potential of Nanotherapies In Diagnosing, Treating Rare CNS Diseases
7 Cyclo Therapeutics Presents Positive Data from Clinical Development Program for Lead Candidate, Trappsol® Cyclo™, at WORLDSymposium 2021
8 Niemann-Pick Disease Type C Therapeutics Market By 2030 Top Winning Strategies, COVID-19 Impacting Factors, Business Strategies | Future Market Insights
9 Heartbroken mum loses all three children to rare Niemann-Pick disease only ever found in 110 Brits
10 Cyclo Therapeutics Unveils New Corporate Identity and Reaffirms Commitment to Improving Quality of Life and Providing Hope for Patients and Families
11 Positive Data Reported in a Multinational Clinical Trial Investigating New Treatment of Niemann-Pick disease Type C
12 Washington mum who lost all three of her children to rare condition on her devastating loss
13 Synaptogenix to Present at Upcoming Scientific Industry Conferences
14 Cat Lawyer Uses Viral Internet Fame to Lend Nonprofit Support
15 IntraBio Expands Niemann-Pick Trial to ID Neuroprotective, Disease-Modifying Effects
16 CytRx Issues Statement Regarding U.S. Regulatory Review of Arimoclomol for Niemann-Pick Disease Type C
17 CytRx Announces Orphazyme's U.S. Expansion Ahead of Potential Regulatory Approval of Arimoclomol for Niemann-Pick Disease Type C
18 Orphazyme submits European Marketing Authorisation Application for arimoclomol for treatment of Niemann-Pick disease Type C
19 Capital increase of 170131 shares in Orphazyme A/S as a result of vesting and exercise of Matching Shares
20 Orphazyme announces U.S. FDA acceptance and Priority Review of New Drug Application for arimoclomol for Niemann-Pick disease Type C
21 Cyclo Therapeutics: A Potential Gem In The Fight Against Alzheimer's Disease
22 Orphazyme completes rolling submission of New Drug Application to U.S. FDA for arimoclomol for Niemann-Pick disease Type C
23 Synaptogenix Files Application to List on the Nasdaq Stock Market
24 Capital increase of 80,553 shares (equivalent to approximately 0.23% of the existing shares) in Orphazyme A/S as a result of an issue of bonus shares to KUMCRI and directed issuance of new shares
25 Cyclo Therapeutics Inc. Announces Positive Safety Profile of Its Drug Trappsol® Cyclo™ in the Treatment of Niemann Pick Disease Type C
26 New report gives national voice to patients, families affected by Niemann-Pick Type C
27 Evox has big pharma partners. Now it has $95M to bring its own drugs to the clinic
28 Brave little Derby girl still smiling despite incurable illness
29 Orphazyme's arimoclomol to be used with Zavesca after FDA approval
30 Niemann-Pick Disease Type C (NPC) Insights, Epidemiology, and Market Forecasts, 2028
31 Evox's exosome platform draws new investors in $95.4M series C
32 CytRx Highlights Orphazyme's Submission of European Marketing Authorisation for Arimoclomol to Treat Niemann-Pick Disease Type C
33 Comparing Cognitive Profiles of Niemann-Pick Disease Type C and Primary Dementia
34 Scenic Biotech Appoints Dr. Philippe Dro as Independent Chairman
35 Mum opens up on losing 3 kids to killer disease so rare only 110 people have ever had it
36 What Is Niemann-Pick Disease?
37 Cyclo Therapeutics Phase 3 Pivotal Program Can Begin Enrollment Per US FDA
38 M6P Therapeutics Announces Formation of Distinguished, Experienced Scientific Advisory Board
39 Potential treatment for Niemann-Pick type C disease
40 NIH teams with industry to develop treatments for Niemann-Pick Type C disease.
41 Cyclo Therapeutics Announces Presentations on Phase I and Phase I/II Trials using Trappsol® Cyclo™ Intravenously to Treat Patients with Niemann-Pick Disease Type C1 at National Niemann Pick Disease Foundation Family Support and Medical Conference
42 Orphazyme's Rare Lipid Disorder Drug Gets Priority Review from the FDA
43 Cyclo Therapeutics Announces Positive Efficacy Data from Extension Protocol with Trappsol® Cyclo™ in Patients with Niemann-Pick Disease Type C
44 Cyclo Therapeutics Inc. Announces Positive Top Line Results from Phase I Trial and Interim Analysis of Phase I/II Trial using Trappsol® Cyclo™ Intravenously to Treat Patients with Niemann-Pick Disease Type C1 (NPC1)
45 Austin Family Battles Rare Disease Amid COVID-19
46 Global Arimoclomol Emerging Insights and Market Forecast Report 2021-2030
47 Orphazyme provides regulatory update on arimoclomol for NPC
48 Effective treatment for Niemann Pick Type C identified
49 Financial calendar 2021 Copenhagen Stock Exchange:ORPHA
50 WGC-Workday at Concession 2021 experts picks: Why we're betting on a Bryson bounce back
51 Mallinckrodt's Niemann-Pick Type C drug fails to hit trial target
52 Cyclo Therapeutics to Present at 2020 Annual Conference for NPC Patients, Families, and Health Professionals in the United Kingdom
53 Experimental treatment for Niemann-Pick disease type C1 appears safe, effective
54 Gene therapy shows promise for treating Niemann-Pick disease type C1
55 Cyclo Therapeutics to Present on Its Clinical and Drug Development Program for Lead Candidate, Trappsol® Cyclo™, at WORLDSymposium 2021
56 Parents of twins with Niemann-Pick find a treatment
57 Niemann-Pick Disease Type C Analysis, Market Size, Epidemiology, Leading Companies and Competitive Analysis By DelveInsight
58 CytRx is Added to the LD Micro Index
59 Family of toddler with Niemann-Pick Type C hopes for treatment
60 Takeda, Evox link up to the tune of $882m
61 Patient Files: Niemann-Pick type C
62 Global Niemann-Pick Disease Type C (NPC) Epidemiology Forecast to 2027, Covering the U.S., EU5 & Japanese Markets
63 Mallinckrodt And VTS-270 In Niemann-Pick Disease Type C: The Competition
64 Sports agate — Feb. 25, 2021
65 Orphazyme accelerates arimoclomol pre-launch activities and updates financial outlook for 2020
66 Orphazyme reports positive arimoclomol data from open-label phase 2/3 extension in Niemann-Pick disease Type C
67 Nearly two years after their narrow PhIII failure, Orphazyme gets speedy look at the FDA
68 ‘Positive and hopeful:’ Greenwich family adapts its fight against rare disorder
69 Idiopathic Hypersomnia Treatment Market Breakdown, Development and New Market Opportunities & Forecasts | Takeda Pharmaceutical Company Limited, Teva Pharmaceutical Industries Ltd., Jazz Pharmaceuticals, Inc
70 Drug trial shows promise for deadly neurological disorder
71 Update: Twins who were face of controversial rare disease treatment have died
72 Why are people fighting over a promising treatment for a fatal childhood disease?
73 Orphazyme A/S provides update on previously announced global offering
74 Cryo-EM structures of NPC1L1 reveal mechanisms of cholesterol transport and ezetimibe inhibition
75 Genesis Invitational 2021 experts picks: Our RED-HOT handicappers are all in on Bryson
76 CytRx Corporation Highlights Arimoclomol Licensee Orphazyme A/S Prepares for Regulatory Filing of Arimoclomol in US for Niemann-Pick Disease Type C
77 IPO Launch: Orphazyme A/S Finalizes Terms For $100 Million U.S. IPO
78 Orphazyme announces presentations at WORLDSymposium™ Copenhagen Stock Exchange:ORPHA
79 Nick Jacobs examines who Chiefs should re-sign, move on from for 2021
80 Ohio 2-year-old battling rare Niemann-Pick disease, dubbed childhood Alzheimers
81 Cyclo Therapeutics Announces Two Presentations at WORLDSymposium2021
82 Niemann-Pick Disease
83 Combination therapy a potential strategy for treating Niemann Pick disease
84 Kim Stratton, CEO, resigns from Orphazyme Copenhagen Stock Exchange:ORPHA
85 Greenwich family takes steps to fight rare childhood disorder
86 Cyclodextrins Market Study Based on Shares, Current Opportunities with Future Growth Scenario by 2026 – The Bisouv Network
87 Comments on: Evox and Takeda sign deal to collaborate on rare disease treatments
88 Cyclo Therapeutics Signs Agreement with Worldwide Clinical Trials to Conduct Pivotal Trial in Niemann-Pick Disease Type C
89 Orphazyme announces Interim Report First Half 2020 Copenhagen Stock Exchange:ORPHA
90 A new therapeutic target against diseases caused by lipid accumulation in cells
91 Michael N. Trinh Named 2020 Merton Bernfield Awardee
92 CTD Holdings Announces FDA Approval of US Expanded Access Treatment Program in Niemann-Pick Disease Type C
93 Orphazyme to take part in panel discussion at the LifeSci Partners 10th Annual Healthcare Corporate Access Event
94 Addi and Cassi Hempel, Reno twins with rare genetic disease whose parents fought FDA, die
95 Arimoclomol Shows Potential to Aid Gaucher Patients in Phase 2 Trial
96 Drug reformulation may improve treatment of Niemann-Pick type C disease [PreClinical]
97 Scenic Biotech Harnessing Genetic Suppression to Treat Rare Disease
98 Orphazyme's Drug for Rare Metabolic Disease Aces Clinical Trial
99 CTD Reports Initial Data for Two Clinical Trials of Trappsol® Cyclo™ Provided Intravenously for Patients with Niemann-Pick Disease Type C
100 Orphazyme announces Annual Report 2019 Copenhagen Stock Exchange:ORPHA