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uniQure Announces Long-Term Clinical Data from Ongoing Phases and Confirms Dose for Hemophilia B Study

December 5, 2018

The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure N.V., a gene therapy company advancing transformative therapies for patients with severe medical needs, announced updated results from its ongoing Phase I/II trial of AMT-060, and provided an update on AMT-061, the company’s next-generation gene therapy candidate […]

Update on uniQure’s Hemophilia B Gene Therapy Program

November 28, 2018

The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure announces the initial topline data from our Phase 2B dose-confirmation study of AMT-061, an investigational gene therapy for the treatment of patients with severe and moderately severe hemophilia B. All patients achieved and sustained therapeutic […]

uniQure Announces First Patient Treated in Dose-Confirmation Study in Patients with Hemophilia B

November 19, 2018

The following is an excerpt from a press release from uniQure. uniQure announced that they have treated the first patient in our Phase 2B dose-confirmation study of AMT-061, an investigational gene therapy for the treatment of patients with severe and moderately severe hemophilia B (see attached press release). “As a one-time administered therapy, AMT-061 has the […]

uniQure Announces First Patient Treated in Dose-Confirmation Study in Patients with Hemophilia B

August 24, 2018

The following is an excerpt from a press release from uniQure. Read the press release in its entirety here.  uniQure, a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, announced it has treated the first patient in its Phase IIb dose-confirmation study of AMT-061, an investigational AAV5-based gene therapy […]

Catalyst Biosciences releases update on trial of Factor IX treatment of severe hemophilia B

June 19, 2018

The following is a summary of a press release from Catalyst Biosciences by Dr. Robert Sidonio, a trusted medical advisor. Read the full press release here. On June 18, Catalyst Biosciences provided an update of the progress from its Phase ½ trial investigating the use of FIX variant product, called CB 2679d/ISU304, which is a […]

Novo Nordisk Launches Rebinyn in the United States for People with Hemophilia B

February 21, 2018

Note: The following is an excerpt from a press release from Novo Nordisk. Read the full press release here.  Novo Nordisk, a global healthcare company, today announced that Rebinyn, Coagulation Factor IX (Recombinant), GlycoPEGylated, is now available in the United States for the treatment of hemophilia B. Rebinyn is an extended half-life injectable medicine used to […]

Breakthrough in Hemophilia Gene Therapy

January 31, 2018

The following is an excerpt from an article in Medical News Bulletin. Read the full article here. Researchers at Spark Therapeutics and Pfizer tested the safety and efficacy of a new hemophilia gene therapy vector for the treatment of hemophilia B. The body’s ability to stop bleeding and repair damaged blood vessels depends heavily upon […]

Catalyst Biosciences Initiates Phase 2 of Potential Hemophilia A and B Therapy

January 5, 2018

Note: The following is an excerpt from an article by Rare Disease Report. Read the full article in its entirety here. On Jan. 4, Catalyst Biosciences announced the initiation and open enrollment of the Phase 2 part of its Phase 2/3 program of marzeptacog alfa (activated) (MarzAA), a highly potent, subcutaneously administered Factor VIIa therapy in development for the treatment […]

uniQure Announces Hemophilia B Gene Therapy Program To Enter Pivotal Study

October 19, 2017

Note: The followed is excerpted from a press release from uniQure. Read uniQure’s full press release HERE.  uniQure announced today plans to advance their development program for hemophilia B gene therapy into a pivotal study using an enhanced version of their product candidate called AMT-061. AMT-061 is identical to AMT-060 in every way apart from a change from […]

Update 2: Bioverativ Files Complaints Against CSL Behring’s Idelvion

July 19, 2017

As noted in our July 10, 2017 story, Bioverativ has filed complaints in the U.S. District Court for the District of Delaware and with the U.S. International Trade Commission alleging that Idelvion, a novel factor IX albumin fusion protein developed by CSL Behring, infringes upon three Bioverativ patents. HFA, in collaboration with the National Hemophilia […]

Spark Therapeutics Presents Updated Interim Hemophilia B Data Supporting Consistent & Sustained Response at ISTH 2017

July 11, 2017

Note: The following is an edited version of a press release from Spark Therapeutics. Read the press release from Spark Therapeutics in it’s entirety here. Spark Therapeutics recently announced that 10 participants in its ongoing Phase 1/2 clinical trial of SPK-9001 for hemophilia B, as of the June 5, 2017 data cut off, had their AIR reduced approximately […]

uniQure Announces Updated, Long-Term Clinical Data from Ongoing Phase I/II Trial of AMT-060 In Patients with Severe Hemophilia B

July 11, 2017

Note: The following is an edited version of a press release from uniQure. Read the press release from uniQure in it’s entirety here. uniQure N.V. has announced updated results from its ongoing, dose-ranging Phase I/II trial of AMT-060, its proprietary, investigational gene therapy in patients with severe hemophilia B. The data includes up to 18 months of follow-up from the low-dose […]

Bioverativ Files Complaints Against CSL Behring’s Idelvion

July 10, 2017

Note: This story has been sourced from the website, www.thepharmaletter.com and The Australian Business Review. On Friday, July 7, 2017, Bioverativ filed complaints in the U.S. District Court for the District of Delaware and with the U.S. International Trade Commission alleging that Idelvion, a novel factor IX albumin fusion protein developed by CSL Behring, infringes upon […]

FDA Approves New Novo Nordisk Treatment for Patients with Hemophilia B

June 1, 2017

  Novo Nordisk recently announced that the U.S. Food and Drug Administration (FDA) has approved the Biologics License Application for REBINYN® (Coagulation Factor IX (Recombinant), GlycoPEGylated) for the treatment of adults and children with hemophilia B. Novo Nordisk expects to launch REBINYN® in the U.S. in the first half of 2018. For more info on REBINYN, […]

uniQure Receives EU PRIME Designation For Hemophilia B Product

May 2, 2017

 uniQure recently received the designation of a “priority medicine” from the European Union for a potential gene therapy treatment for Hemophilia B. From the press release: uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that AMT-060, its proprietary, investigational gene therapy in patients with severe […]

Catalyst Biosciences Completes New Hemophilia B Drug Toxicology Study

April 14, 2017

On April 11, Catalyst Biosciences, a clinical-stage biopharmaceutical company focused on developing novel medicines to address hematology indications, reached a key milestone towards starting human trials with completion of the CB 2679d/ISU304 toxicology studies. Their press release can be read in full here.

Aptevo’s Ixinity Found To Be Safe In Children Under 12 With Hemophilia B

April 14, 2017

Aptevo’s Ixinity, previously approved for use in people over the age of 12, just received approval for use in children under 12, per their ongoing phase 3 trial. Read the full press release here.

Novo Nordisk Announces New Data on Psychosocial Impact of People Living with Hemophilia B

July 26, 2016

Note: The below is an edited version of a press release by Novo Nordisk. The original release can be read in it’s entirety here.  Novo Nordisk today announced the first results from the Bridging Hemophilia B Experiences, Results and Opportunities into Solutions (B-HERO-S) study, which found that many adults and children living with even mild or […]

Spark Therapeutics and Pfizer Announce Receipt of FDA Breakthrough Therapy Designation for SPK-9001 for the Treatment of Hemophilia B

July 21, 2016

Note: The below is an edited version of a press release by Spark Therapeutics. The original release can be read in it’s entirety here. Spark Therapeutics and Pfizer Inc. announced today that the U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to SPK-9001, the lead investigational candidate in the companies’ SPK-FIX program, in development for the treatment of hemophilia B. SPK-9001, a […]

uniQure Announces Data from Phase I/II AMT-060 Clinical Trial

June 28, 2016

Note: The below is an edited version of a press release by UniQure. The original release can be read in it’s entirety here. On June 11, 2016, uniQure N.V. announced that additional data from its Phase I/II clinical trial of AMT-060 in hemophilia B patients were presented at the 21st Congress of the European Hematology Association (EHA) in Copenhagen, […]

Spark Therapeutics Announces Updated Data from Hemophilia B Trial

June 14, 2016

Note: The below is an edited version of a press release by Spark Therapeutics. The original release can be read in it’s entirety here. PHILADELPHIA, June 13, 2016 – Spark Therapeutics announced today updated results of the first cohort from the ongoing Phase 1/2 clinical trial of SPK-9001, the lead investigational candidate in its SPK-FIX program, which is being studied for the […]

Trial for SubQ Hemophilia Inhibitor Treatment Begins

March 14, 2016

Note: The below is an edited press release from Alnylam Pharmaceuticals, Inc. The original release can be read in its entirety here.  Alnylam Pharmaceuticals, Inc. announced on Monday, March 14, 2016, that it has initiated dosing of hemophilia patients with inhibitors in Part D of an ongoing Phase 1 clinical trial evaluating a once-monthly subcutaneous dose […]

FDA Approves First 14-Day Hemophilia B Treatment

March 5, 2016

Note: The following is an edited form of a press release from the FDA . The original form of the release can be read here. The U.S. Food and Drug Administration approved on Friday, March 4, Idelvion, Coagulation Factor IX (Recombinant), Albumin Fusion Protein, for use in children and adults with Hemophilia B. Idelvion is the […]

HFA Observes February 29 As Rare Disease Day

February 23, 2016

The Hemophilia Federation of America (HFA) joins the National Organization for Rare Disorders (NORD) on February 29, and others around the world in observing Rare Disease Day. The purpose of this day is to focus attention on the needs of patients and families affected by rare diseases. In the U.S., any disease affecting fewer than […]

Dear Addy: Why Are Medical Alert IDs Important?

January 15, 2016

Dear Addy, Several parents that I’ve met at the HTC have recommended I should get my daughter a medical ID bracelet. Why is this so important? Are there resources available to help me get her one? Signed Inquiring About IDs Dear Inquiring, For patients with serious medical conditions like a bleeding disorder, it’s important that […]

Dimension Therapeutics Announces Initiation of Phase 1/2 Study For Hemophilia B Treatment

January 7, 2016

Note: The below is an edited press release from Dimension Therapeutics, Inc. The original release can be read here. Dimension Therapeutics, Inc. announced on January 7 the initiation of a phase 1/2 study of DTX101, the company’s lead therapeutic candidate, for the treatment of patients with hemophilia B. DTX101 is designed to deliver stable expression […]

uniQure Announces Preliminary Results from Hemophilia B Gene Therapy Trial

January 7, 2016

Note: the following is the  edited form of a  press releaseoriginally published by uniQure. The full release can be read here.  On Thursday, January 7, 2016, uniQure N.V. announced preliminary topline results from the low-dose cohort of an ongoing Phase I/II clinical trial being conducted in adult hemophilia B patients treated with uniQure’s novel AAV5/FIX […]

Phase I Study Results for Once-Monthly Subcutaneous Hemophilia Treatment

December 8, 2015

Note: The following is an edited press release from Alnylam. The original release can be read in its entirety here. Alnylam Pharmaceuticals, Inc. announced on December 7, 2015 positive results from its ongoing Phase 1 clinical study with fitusiran (fi-TOO-si-ran), the recommended International Nonproprietary Name (INN) for ALN-AT3. Fitusiran is an investigational RNAi therapeutic targeting […]

New Data Released on Long-Acting Hemophilia B Treatment

December 8, 2015

Note: The following is the edited version of a press release issued by CSL Behring. The original release can be found here. On December 7, 2015, CSL Behring presented data from its Phase III PROLONG-9FP clinical program evaluating the efficacy and long-term safety of its investigational long-acting fusion protein linking recombinant coagulation factor IX with recombinant […]

Pain and Mobility Are Major Problems for Patients with Hemophilia

December 8, 2015

Note: The following originally appeared on MDMagazine.com. New research further supports the understanding that patients with hemophilia suffer from pain and mobility issues, and it went on to uncover which joints are most greatly affected by the condition. Christine Kempton, MD, MSc, from Emory University in Georgia, and colleagues used multiple strategies to find how […]


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