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hemophilia

Blood Cell Traits Linked to Individual Loci, Polygenic Contributors in International Studies

Two new papers have identified thousands of genetic loci with ties to more than two-dozen blood cell traits in individuals from up to five ancestry groups.

Asks for More

Reuters reports that the Food and Drug Administration has rejected BioMarin's gene therapy for hemophilia A and has asked for longer-term data.

A High Price

A gene therapy for hemophilia could cost millions of dollars, NPR writes.

New Hemophilia Treatment Data

Spark Therapeutics reports new data on its investigational hemophilia A treatment that says it reduces bleeds, according to Stat News.

Quickly, Quickly

Gene therapies could qualify for a faster US Food and Drug Administration approval process, according to Stat News.

Oct 9, 2014

Alnylam Sees a Return to Big Indications amid Advances in Rare Diseases
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Aug 7, 2014

Alnylam Hemophilia Drug Gets European Orphan Drug Status
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Jan 23, 2014

Alnylam Begins Phase I Testing of Hemophilia Drug
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Oct 31, 2013

Alnylam Files to Begin Phase I Trial of Hemophilia Drug
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Oct 9, 2013

Hemophilia Genotyping Effort Funded by Biogen Idec Expanded Nationwide
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Aug 22, 2013

Alnylam Receives Additional Orphan Drug Designation for Hemophilia Drug
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Aug 15, 2013

Alnylam Execs Highlight Conjugate Delivery Tech as Lipid-Enabled TTR Drug Advances
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Aug 15, 2013

Alnylam Receives Orphan Drug Designation for Hemophilia B Candidate
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Jul 3, 2013

Alnylam Releases Positive Preclinical Data from Hemophilia Program
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May 9, 2013

Alnylam to Release Phase II ATTR Data, Launch Expansion Trial in Coming Months
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Jan 10, 2013

Alnylam Adds New Program to Pipeline as It Shelves Anemia Candidate
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Dec 13, 2012

Alnylam Presents Preclinical Data from Hemophilia, Hemoglobinopathy Programs
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Nov 14, 2012

Biogen Idec and Partners to Offer Free Genetic Testing to Hemophilia Patients to Advance Insights
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Nov 8, 2012

Biogen Idec, NHF Launch Hemophilia Genotype Testing Initiative

Nov 8, 2012

Alnylam ATTR Program on Track as Company Explores Subcutaneous Delivery for Cholesterol Drug
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Aug 9, 2012

Reporting Positive Data on Conjugate Delivery Tech, Alnylam Preps IND for Subcutaneous ATTR Therapy
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Jul 12, 2012

Alnylam Selects Hemophilia Candidate, Releases Preclinical Data
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Feb 16, 2012

Alnylam Provides Timelines for Drug Programs, Offers No Formal Guidance on Partnerships
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Jan 12, 2012

Alnylam Focuses on Two Lead Programs, Won't Advance Others without Partners
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Sep 16, 2011

Stanford-Led Team Demonstrates Utility of Ethnicity-Specific Reference for Interpreting Genome Data

Breaking News
The Scan

Two J&J Doses

Johnson & Johnson says two doses of its SARS-CoV-2 vaccine provides increased protection against symptomatic COVID-19, CNN reports.

Pfizer-BioNTech Vaccine Response in Kids

The Pfizer-BioNTech SARS-CoV-2 vaccine in a lower-dose format appears to generate an immune response among children, according to the Washington Post.

Chicken Changes to Prevent Disease

The Guardian writes that researchers are looking at gene editing chickens to help prevent future pandemics.

PNAS Papers on Siberian Dog Ancestry, Insect Reproduction, Hippocampal Neurogenesis

In PNAS this week: ancestry and admixture among Siberian dogs, hormone role in fruit fly reproduction, and more.