News

BioMarin Hemophilia A Clinical Development Program: An Update for the Hemophilia Community
Industry News & Research
BioMarin is committed to updating the community regarding our ongoing gene therapy clinical trial program in hemophilia A. BioMarin’s investigational gene therapy…

FDA Recommends Additional Data for BioMarin’s Investigational Gene Therapy
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
Biomarin announced that they received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) on August 18th for their…

Novo Nordisk Announces Near Resumption of Concizumab Trials
Industry News & Research
Novo Nordisk has announced that clinical trials for their investigational subcutaneous therapy concizumab will resume after an approximate five month pause, initiated…

Medical Advisory #427: Stimate Recall Update
Industry News & Research
August 13, 2020 CSL Behring and Ferring Pharmaceuticals have announced additional information about a recent recall of lots of Stimate® that was…

A New Study of Hemophilia Occurrence Finds Many More Cases in the United States
Industry News & Research
Hemophilia is an inherited bleeding disorder in which the blood does not clot properly due to a lack or decrease in a…

MASAC Encourages People with Bleeding Disorders to be Included in COVID-19 Vaccine Trials
COVID-19, Industry News & Research
Individuals with inherited and acquired bleeding disorders, including hemophilia have substantial deficits in coagulation, for which they receive a variety of protein…

New Online Space for Black/African Americans with Bleeding Disorders
Industry News & Research
In 2019, the National Hemophilia Foundation conducted a series of listening sessions and guided conversations with Black and African American members of…

Stimate Recall Update – CSL Behring & Ferring Pharmaceuticals Response to HFA-NHF Letter, Voluntary Recall Due to “Superpotency”
Advocacy & Legislation, Industry News & Research
Ferring Pharmaceuticals issued a voluntary recall of Stimate (desmopressin) nasal spray due to “superpotency” – in other words, amounts of desmopressin being higher than…

HFA and NHF Respond to Stimate Drug Recall
Industry News & Research
On July 21, CSL Behring and Ferring Pharmaceuticals announced a recall of multiple lots of Stimate (desmopressin) in the United States and in other countries…

Alex Borstein Brings Star Power to Raise Awareness of VWD
Industry News & Research
The National Hemophilia Foundation (NHF) and Takeda have teamed up to launch a series of PSAs and resources that hope to increase…

A New Path Forward in Research
Industry News & Research
At the National Hemophilia Foundation’s (NHF) Bleeding Disorders Conference, held virtually this year, NHF president and CEO Leonard Valentino, MD, asked each…

Establishing a New Paradigm to Realize Health Equity in Hemophilia
Industry News & Research
A paper published in the journal Haemophilia, suggests that clinical expectations for people with hemophilia have remained relatively static for years, falling…

Irish Investigators Review Fetal Precautions and Delivery Mode in Congenital Bleeding Disorders
Industry News & Research
It is well established that the clinical management of pregnant women with congenital bleeding disorders (CBDs) is challenging, as healthcare providers, including…

Stimate Recall: NHF and HFA Seek Answers
Advocacy & Legislation, Industry News & Research
Immediately following the issuance of medical advisories to the community regarding the recall of Stimate® (desmopressin nasal spray ) a product used to control…

NHF Medical Advisory: CSL Behring and Ferring Pharmaceuticals have announced a recall of lots of Stimate®
Industry News & Research
Ferring Pharmaceuticals Inc. is initiating a precautionary voluntary recall of all batches of Stimate® Nasal Spray (desmopressin acetate) 1.5 mg/mL. During testing…

New Data From Phase IIIb Study Reinforces Safety Profile of Genentech’s Hemlibra (emicizumab-kxwh) in People With Hemophilia A
Industry News & Research
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced results from the second interim analysis of the…

New Partners Module Explores Home Treatment Logs
Industry News & Research
The Partners in Bleeding Disorders Education Program has announced the launch of a new online learning module, “Accounting for Home Care: The Role…

CSL Acquires Hemophilia B Gene Therapy Candidate from uniQure
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
CSL Behring recently announced that it has agreed to acquire exclusive global license rights from uniQure, to commercialize etranacogene dezaparvovec (AMT-061), an…

Sanofi and Sobi Make Additional Factor Donation to Aid Program
Industry News & Research
Sanofi and Sobi, along with the World Federation of Hemophilia (WFH) and WFH USA recently announced as extension of the two companies’…

Sanofi and Sobi Make Additional Factor Donation to Aid Program
Industry News & Research
Sanofi and Sobi, along with the World Federation of Hemophilia (WFH) and WFH USA recently announced as extension of the two companies’…

Hemophilia History the Focus of New Partners Modules
Industry News & Research
The Partners in Bleeding Disorders Education Program has announced the launch of two new online learning modules that speak to the history of…

BioMarin Releases Trial Updates in Lead Up to WFH Virtual Summit
Industry News & Research
BioMarin has released updates on the ongoing clinical trial program for their investigational gene therapy valoctocogene roxaparvovec, which is intended for the…

Investigators Look at Hematuria as Hypertension Risk Factor in Hemophilia Patients
Industry News & Research
Findings from an international study published in the journal Medicine suggest that the visible presence of blood in urine (macroscopic hematuria) could represent increased…

Judith Graham Pool Postdoctoral Research Fellowships
Industry News & Research
Letter of Intent Deadline: July, 5, 2020 Application Deadline: August 16, 2020 Award Start Date: December 2020 NHF’s premier research fellowship program is named in honor…