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BioNews, parent company of Acute Myeloid Leukemia News, invites the members of our rare disease communities to join us i...
23/01/2023

BioNews, parent company of Acute Myeloid Leukemia News, invites the members of our rare disease communities to join us in recognizing Rare Disease Day in February by answering the question, “Why does rare disease awareness matter to you?” Email your 150-200-word response to [email protected] along with two high-quality pictures of yourself that capture your “why.” We encourage you to share your response on your personal social media accounts tagging BioNews and using the hashtag . Throughout February, your submission could be featured on our social media channels! More info: https://buff.ly/3GTFXxH

CRISPR Gene Editing Used to Build 1st Model of AML Progression
22/02/2021

CRISPR Gene Editing Used to Build 1st Model of AML Progression

Blocking early inflammatory and innate immune signaling is a promising therapeutic target in acute myeloid leukemia (AML), as disruptions in these signaling pathways occur early and persist throughout the evolution of the disorder. This result comes from a recent study, in which researchers used...

Potential Therapy May Better Prepare Older Patients Before Stem Cell Transplant
18/02/2021

Potential Therapy May Better Prepare Older Patients Before Stem Cell Transplant

JSP191, an investigational therapy by Jasper Therapeutics, showed promising efficacy as a conditioning agent to prepare older patients with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) for a stem cell transplant. These are the early findings of an ongoing open-label Phase 1...

Eurodis Survey: Healthcare Experience Worse for Rare Disease Patients
16/02/2021

Eurodis Survey: Healthcare Experience Worse for Rare Disease Patients

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of...

Eurodis Survey: Healthcare Experience Worse for Rare Disease Patients
16/02/2021

Eurodis Survey: Healthcare Experience Worse for Rare Disease Patients

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FDA Grants Iadademstat Orphan Drug Designation
15/02/2021

FDA Grants Iadademstat Orphan Drug Designation

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Oryzon Genomics' iadademstat, an investigational oral therapy for people with acute myeloid leukemia (AML). “Receiving Orphan Drug Designation for iadademstat in AML is an important recognition of the role that...

Eprenetapopt-Azacitidine Combo Shows Promise for AML With TP53 Mutations
12/02/2021

Eprenetapopt-Azacitidine Combo Shows Promise for AML With TP53 Mutations

Eprenetapopt (APR-246), Aprea Therapeutics’ lead cancer therapy candidate, was well-tolerated and showed promising clinical activity when given alongside azacitidine to treat patients with myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML) with mutations in the tumor suppressor...

Pandemic Won’t Stop Rare Disease Day on Feb. 28
11/02/2021

Pandemic Won’t Stop Rare Disease Day on Feb. 28

Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport...

Pandemic Won’t Stop Rare Disease Day on Feb. 28
11/02/2021

Pandemic Won’t Stop Rare Disease Day on Feb. 28

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Xospata Conditionally Approved in China for Certain Acute Myeloid Leukemias
08/02/2021

Xospata Conditionally Approved in China for Certain Acute Myeloid Leukemias

Xospata (gilteritinib) has been conditionally approved in China to treat adults who have relapsed or refractory acute myeloid leukemia (AML) with mutations in the FLT3 gene. The therapy’s conditional approval by the Chinese National Medical Products Administration (NMPA) followed an expedited...

New Combo Therapy Shows Promising Activity Against AML in Lab Studies
04/02/2021

New Combo Therapy Shows Promising Activity Against AML in Lab Studies

Combining two potential cancer therapies with distinct modes of action and limited clinical benefits on their own resulted in powerful anti-cancer activity in both cellular and mouse models of acute myeloid leukemia (AML), a study shows. Notably, these benefits were found to be associated with...

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns
03/02/2021

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a "State Report Card" argues that many concerns — notably out-of-pocket costs for prescription medicines and access to affordable comprehensive care — still need attention. Those...

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns
03/02/2021

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

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TXNIP Protein Deficiency Linked to Poor Prognosis for AML Patients, Study Finds
12/10/2020

TXNIP Protein Deficiency Linked to Poor Prognosis for AML Patients, Study Finds

Low expression levels of the protein TXNIP — thioredoxin interacting protein — was associated with worse outcomes in people with acute myeloid leukemia (AML), according to the results of a new study. Artificially increasing the amount of this protein prevented AML cells from growing in a...

Phase 1 Trial Finishes Dosing First Group of AML Patients With Actimab-A
09/10/2020

Phase 1 Trial Finishes Dosing First Group of AML Patients With Actimab-A

The first group of patients with relapsed or refractory acute myeloid leukemia (AML) has been dosed in a Phase 1/2 dose-escalation trial assessing the safety and efficacy of Actimab-A and Venclexta (venetoclax) as a combination therapy, Actinium Pharmaceuticals announced. Participants receiving...

Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos
08/10/2020

Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos

Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare diseases, including amyotrophic lateral sclerosis (ALS). The organization’s panel of judges has pared the number.....

Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos
08/10/2020

Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos

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‘Find the Right Fit’ Aims to Educate, Support People With Secondary AML
01/10/2020

‘Find the Right Fit’ Aims to Educate, Support People With Secondary AML

Jazz Pharmaceuticals, with help from the Myelodysplastic Syndrome (MDS) Foundation and the Cancer Support Community, has launched Find the Right Fit, an initiative providing educational resources about MDS and secondary acute myeloid leukemia (sAML). An initiative goal is to help people recently...

First Clinical Trial Launched for AML, Other Blood Cancer Patients With COVID-19
28/09/2020

First Clinical Trial Launched for AML, Other Blood Cancer Patients With COVID-19

The Leukemia & Lymphoma Society (LLS) has announced the launch of the first clinical trial dedicated to people with blood cancers, including acute myeloid leukemia (AML), who are infected with COVID-19. The study will be conducted as part of the Beat AML Master Clinical Trial (NCT03013998), a...

Dosing Begins in Phase 1 Trial Testing Immune-Onc’s IO-202 for Advanced AML, CMML
24/09/2020

Dosing Begins in Phase 1 Trial Testing Immune-Onc’s IO-202 for Advanced AML, CMML

The first patient has been dosed in Immune-Onc Therapeutic’s Phase 1 clinical trial of IO-202, its lead antibody treatment candidate for advanced acute myeloid leukemia (AML) and chronic myelomonocytic leukemia (CMML). The trial (NCT04372433), underway at City of Hope, in California, and at the...

Rigosertib Combo Fails to Prolong Survival in Higher-risk Myelodysplastic Syndromes
21/09/2020

Rigosertib Combo Fails to Prolong Survival in Higher-risk Myelodysplastic Syndromes

When given alongside best supportive care, rigosertib failed to prolong the survival of patients with higher-risk myelodysplastic syndromes (HR-MDS), a group of blood cancers that often progress to acute myeloid leukemia (AML), a Phase 3 trial has found. The main goal of the INSPIRE study...

FDA Grants Rare Pediatric Disease Status to AML Therapy OXi-4503
17/09/2020

FDA Grants Rare Pediatric Disease Status to AML Therapy OXi-4503

The U.S. Food and Drug Administration (FDA) has granted its Rare Pediatric Disease designation to Mateon Therapeutics’ OXi-4503 (combretastatin A1-diphosphate, CA1P) for the treatment of children and teens with acute myeloid leukemia (AML) who carry genetic mutations that disproportionately...

French Authorities Advance Phase 3 Trial Testing Sellas’ GPS Cancer Vaccine
14/09/2020

French Authorities Advance Phase 3 Trial Testing Sellas’ GPS Cancer Vaccine

French regulatory authorities have approved an investigational medicinal product dossier (IMPD) from Sellas Life Sciences, granting permission to advance in France a Phase 3 clinical trial testing the cancer vaccine galinpepimut-S (GPS) in people with acute myeloid leukemia (AML). The REGAL...

LLS Highlights Its Support Programs During Blood Cancer Awareness Month
10/09/2020

LLS Highlights Its Support Programs During Blood Cancer Awareness Month

Throughout September for Blood Cancer Awareness Month, the Leukemia & Lymphoma Society (LLS) is asking the public to join its commitment to research, education, advocacy, and patient support. Blood cancers, including acute myeloid leukemia, are diagnosed every three minutes in the U.S. Those who...

LLS Highlights Its Support Programs During Blood Cancer Awareness Month
09/09/2020

LLS Highlights Its Support Programs During Blood Cancer Awareness Month

No Description.

FDA Approves Oral Chemotherapy Onureg for Newly Diagnosed AML Patients in Remission
03/09/2020

FDA Approves Oral Chemotherapy Onureg for Newly Diagnosed AML Patients in Remission

The U.S. Food and Drug Administration has approved Onureg, an oral formulation of the chemotherapy azacitidine, as a maintenance treatment for adults with newly diagnosed acute myeloid leukemia (AML) who achieved complete remission after intensive induction chemotherapy. The approval is for...

Idhifa Combo Fails to Prolong Survival in Older Patients in Phase 3 Trial
01/09/2020

Idhifa Combo Fails to Prolong Survival in Older Patients in Phase 3 Trial

Idhifa (enasidenib), given alongside best supportive care, failed to prolong overall survival of older adults with relapsed or refractory acute myeloid leukemia (AML) carrying a mutation in the IDH2 gene, Phase 3 trial data show. The main goal of the study, called IDHENTIFY (NCT02577406), was to...

Same But Different Photo Contest Celebrates People With Rare Diseases
31/08/2020

Same But Different Photo Contest Celebrates People With Rare Diseases

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme "A Glimmer of Hope," the competition is a means to "visually express the hope that exists for people affected...

Venclexta Plus Vidaza Increases AML Survival, Study Shows
27/08/2020

Venclexta Plus Vidaza Increases AML Survival, Study Shows

Adding Venclexta (venetoclax) to treatment with Vidaza (azacitidine) significantly prolongs survival in people with acute myeloid leukemia (AML) who have not been treated before, a new study demonstrates. The study, "Azacitidine and Venetoclax in Previously Untreated Acute Myeloid Leukemia," was...

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input
24/08/2020

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new...

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