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Tag: genetic disease

CTD Holdings Presents Compassionate Use Data on Trappsol(R) Cyclo(TM) to Treat Niemann-Pick Disease Type C and Provides Update on Progress of U.S. and EU Clinical Trials at 13th Annual WORLDSymposium

CTD Holdings, Inc. (OTCQB: CTDH), a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of disease, presented updated compassionate use data regarding the Company’s proprietary formulation of hydroxypropyl beta cyclodextrin, Trappsol® Cyclo™, for the treatment of Niemann-Pick Disease Type C (NPC). The data were presented in a poster

Predictive Technology Group, Inc. (PRED) Announces Acquisition of US Patent Application for Method for Treating Patients with Stem Cells “Cleaned” via CRISPR

Predictive Technology Group, Inc. (OTC PINK: PRED), a life sciences technology holding company, announced the acquisition of US patent application 62/295,042 direct to the practice of “cleaning” stem cells of a predetermined disease associated marker via the “Clustered Regularly Interspaced Short Palindromic Repeats –CRISPR” process and administering the stem cells

Wilson Therapeutics Announces Support of Big Wow

Wilson Therapeutics AB today announced its sponsorship of the second annual Big Walk on Wilson’s (Big Wow). The event will take place on October 1, 2016, in 24 cities across North America to increase awareness of Wilson Disease and raise funds for the Wilson Disease Association.

Arrowhead Pharmaceuticals Initiates Phase 2 Study of ARC-AAT

PASADENA, Calif.–(BUSINESS WIRE)–Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it
initiated a Phase 2 clinical study of ARC-AAT, an investigational
RNAi-based medicine for the treatment of liver disease associated with
alpha-1 antitrypsin deficiency (AATD). The study is designed to evaluate
safety and tolerability and determine the effect of multiple doses of
ARC-AAT on levels

Concert Pharmaceuticals Presents CTP-656 Phase 1 Results at European Cystic Fibrosis Conference

LEXINGTON, Mass.–(BUSINESS WIRE)–Concert
Pharmaceuticals, Inc. (NASDAQ: CNCE) today presented results from a
Phase 1 multiple ascending dose trial of CTP-656, a next generation CFTR
potentiator being developed for the treatment of cystic fibrosis. The
Phase 1 healthy volunteer trial included a single dose tablet crossover
comparison with Kalydeco®, the current standard of care for certain
gating

Concert Pharmaceuticals Presents CTP-656 Phase 1 Results at European Cystic Fibrosis Conference

LEXINGTON, Mass.–(BUSINESS WIRE)–Concert
Pharmaceuticals, Inc. (NASDAQ: CNCE) today presented results from a
Phase 1 multiple ascending dose trial of CTP-656, a next generation CFTR
potentiator being developed for the treatment of cystic fibrosis. The
Phase 1 healthy volunteer trial included a single dose tablet crossover
comparison with Kalydeco®, the current standard of care for certain
gating

Macrogen Launches famplan™ Genetic Carrier Testing, and ABOOBA™ Genetic Test for Newborns

SEOUL, South Korea–(BUSINESS WIRE)–Macrogen (CEO Hyon-yong Chong, www.macrogen.com)
(KOSDAQ:038290), a biotechnology company in the area of precision
medicine, announced its official worldwide launch of famplan™, a genetic
carrier test, and ABOOBA™, a genetic test for newborns, in celebration
of May 15, the International Day of Families.

famplan™ is an abbreviation for “family plan,” and it

Ionis Pharmaceuticals Reports Data Update from Nusinersen Phase 2 Study in Infants with Spinal Muscular Atrophy and Reviews Neurological Disease Franchise

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today provided an update on its ongoing open-label Phase 2 clinical study of nusinersen in infants with spinal muscular atrophy (SMA) at the American Academy of Neurology (AAN) meeting in Vancouver, BC.  The data reported today show that there have been no new events, as defined

CombiMatrix Launches Preimplantation Genetic Diagnostic Testing for Single Gene Disorders and Chromosomal Translocations

CombiMatrix Corporation (NASDAQ:CBMX), a molecular diagnostics company specializing in DNA-based testing services for prenatal and postnatal developmental disorders and pre-implantation genetic screening services, announces the launch of CombiPGD™ – Preimplantation Genetic Diagnosis (PGD) for single gene disorders and chromosomal translocations.  CombiPGD is a test performed prior to embryo implantation to

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