Scd

Imara Announces Completion of Patient Enrollment in Ardent Phase 2b Clinical Trial of IMR-687 (tovinontrine) for Sickle Cell Disease

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星期四, 八月 5, 2021
Company, U.S. Securities and Exchange Commission, Cell adhesion, Private Securities Litigation Reform Act, Inflammation, Development, Fetal hemoglobin, Security (finance), Patient, Union of South American Nations, Trial of the century, Ardent, Therapy, COVID-19, CGMP, HBF, Thalassemia, Adult, Scd, Nasdaq, PDE9, Sickle cell disease, Safety, Lists of diseases, Goal, GLOBE, Pharmaceutical industry

We are pleased to achieve this important milestone for IMR-687, said Rahul Ballal, Ph.D., President and Chief Executive Officer of Imara.

Key Points: 
  • We are pleased to achieve this important milestone for IMR-687, said Rahul Ballal, Ph.D., President and Chief Executive Officer of Imara.
  • Furthermore, we are excited to have enrolled subjects from across the world, including in Africa, making this a truly global effort.
  • The Ardent Phase 2b clinical trial is a randomized, double-blind, placebo-controlled, multicenter study designed to evaluate the safety and efficacy of IMR-687 administered once daily in approximately 99 adult patients with sickle cell disease.
  • These statements include, but are not limited to, statements relating to the timing with respect to reporting of data from the Ardent Phase 2b clinical trial in patients with sickle cell disease.

Vifor Pharma reports strong H1 2021 growth, on track to meet full year guidance1

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星期四, 八月 5, 2021
Biotechnology, Pharmaceutical, Health, Iron-deficiency anemia, Growth, United States dollar, Manuscript, ANCA, Board, Annual, Marketing, Scd, Income, Hospital, Kidney, Blood, Earnings before interest, taxes, depreciation and amortization, Erythropoiesis-stimulating agent, Review, European Medicines Agency, EMA, Cer, Food and Drug Administration, The New England Journal of Medicine, Sickle cell disease, Therapy, Group, CKD, Anemia, Population, Iron, Mortality, Advisory Committee, European Heart Journal, Depreciation, Dialysis, Raasi, Vifor Pharma, File, Quality of life, Leadership, Hyperkalemia, Awareness, European Space Agency, Pharma, Heart failure, Prescription Drug User Fee Act, Adoption, Annual general meeting, Maa, Executive Committee, Patient, COVID-19, PBM, CHF, NDA, Director, Overalls, Heart, Pharmaceutical industry, Medical device, Aquaculture

Based on the solid results in H1 2021, the Group is confident of achieving its full year guidance.

Key Points: 
  • Based on the solid results in H1 2021, the Group is confident of achieving its full year guidance.
  • Ferinject / Injectafer net sales increased 22.4% (up 22.8% at CER) to CHF 320.5 million in H1 2021 from CHF 261.9 million a year earlier.
  • As a result, Vifor Pharma posted net sales of CHF 73.7 million, up 1.4% or 8.5% at CER.
  • Abbas Hussain was appointed Chief Executive Officer of Vifor Pharma, succeeding Stefan Schulze, who has decided to step down for personal reasons.

GBT Announces Participation at the Wedbush PacGrow Healthcare Conference

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星期三, 八月 4, 2021
GBT, Wedbush Securities

SOUTH SAN FRANCISCO, Calif., Aug. 04, 2021 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc.(GBT) (NASDAQ: GBT) today announced that it will participate in a virtual fireside chat at the Wedbush PacGrow Healthcare Conference on Wednesday, August 11, 2021, at 2:20 p.m.

Key Points: 
  • SOUTH SAN FRANCISCO, Calif., Aug. 04, 2021 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc.(GBT) (NASDAQ: GBT) today announced that it will participate in a virtual fireside chat at the Wedbush PacGrow Healthcare Conference on Wednesday, August 11, 2021, at 2:20 p.m.
  • The fireside chat will be webcast live from GBTs website at www.gbt.com in the Investors section.
  • Global Blood Therapeutics (GBT) is a biopharmaceutical company dedicated to the discovery, development and delivery of life-changing treatments that provide hope to underserved patient communities.
  • Founded in 2011, GBT is delivering on its goal to transform the treatment and care of sickle cell disease (SCD), a lifelong, devastating inherited blood disorder.

Texas Adds Endari® to Latest Preferred Drug List

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星期一, 八月 2, 2021
Healthcare reform in the United States, Health sciences, Pharmaceuticals policy, Health, Federal assistance in the United States, Presidency of Lyndon B. Johnson, Child poverty, Medicaid, Formulary

According to the THHS website, preferred drugs are medications recommended by the Texas Drug Utilization Review Board for their efficaciousness, clinical significance, cost effectiveness, and safety.

Key Points: 
  • According to the THHS website, preferred drugs are medications recommended by the Texas Drug Utilization Review Board for their efficaciousness, clinical significance, cost effectiveness, and safety.
  • The Texas Medicaid Formulary contains all products, including those on the preferred drug list, available to people enrolled in Medicaid.
  • With this recent revision, Texas joins many other states in improving access to Endari for the treatment of sickle cell disease in Medicaid patients.
  • "We greatly appreciate that Texas Health and Human Services has eliminated the prior authorization criteria for Endari.

GBT Provides Regulatory and Pipeline Updates in Sickle Cell Disease (SCD)

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星期四, 七月 22, 2021
Clinical medicine, Health in Africa, Medical specialties, Medical genetics, Hematopathology, Sickle cell disease, Scd, Hematologic disease, Stearoyl-CoA desaturase-1

SOUTH SAN FRANCISCO, Calif., July 22, 2021 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced updates across several of its development programs in sickle cell disease (SCD). The company has submitted a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) seeking accelerated approval for Oxbryta® (voxelotor) for the treatment of SCD in children ages 4 to 11 years, together with a related separate New Drug Application (NDA) required to seek approval for a pediatric weight-based formulation of Oxbryta. Additionally, GBT initiated two global, randomized, placebo-controlled, pivotal Phase 3 clinical trials of inclacumab, a novel P-selectin inhibitor. The company also enrolled the first SCD patient in a Phase 1 study evaluating GBT021601 (GBT601), a next-generation hemoglobin S (HbS) polymerization inhibitor, in people with SCD.

Key Points: 
  • GBT requested priority review for the sNDA and NDA, which, if granted, could result in a six-month review process.
  • Our pipeline is the engine of our commitment to improving the lives of people living with this terrible disease.
  • We are excited to initiate enrollment in our two pivotal studies for inclacumab, in parallel with our Phase 1 study of GBT601 in people with sickle cell disease.
  • Founded in 2011, GBT is delivering on its goal to transform the treatment and care of sickle cell disease (SCD), a lifelong, devastating inherited blood disorder.

Emmaus Life Sciences Announces Submission of Endari® Marketing Authorization Application in Kuwait

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星期四, 七月 15, 2021
Life sciences, Biotechnology, Specialty drugs, Biopharmaceutical, Pharmaceutical industry, Pharmacy

TORRANCE, Calif., July 15, 2021 /PRNewswire/ -- Emmaus Life Sciences, Inc. (OTC: EMMA),a commercial-stage biopharmaceutical company and leader in the treatment of sickle cell disease, announced today the submission of its application for Marketing Authorization (MA) for Endari to the Kuwait Drug and Food Control (KDFC).

Key Points: 
  • TORRANCE, Calif., July 15, 2021 /PRNewswire/ -- Emmaus Life Sciences, Inc. (OTC: EMMA),a commercial-stage biopharmaceutical company and leader in the treatment of sickle cell disease, announced today the submission of its application for Marketing Authorization (MA) for Endari to the Kuwait Drug and Food Control (KDFC).
  • This is the first step in the registration of Endari by the KDFC, which is responsible for the registration of pharmaceutical products in Kuwait.
  • "We are pleased to announce the submission of our marketing authorization application to the Kuwaiti authorities.
  • Emmaus Life Sciences, Inc. is a commercial-stage biopharmaceutical company engaged in the discovery, development, marketing and sale of innovative treatments and therapies, including those in the rare and orphan disease categories.

GBT Awards $450,000 in ACCEL Grants to Improve Access to Care in Sickle Cell Disease

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星期二, 六月 15, 2021
Health in Africa, Hematopathology, Sickle cell disease, Clinical medicine, Medical specialties, Scd, Branches of biology, Stearoyl-CoA desaturase-1

We are confident that the programs proposed by this years grantees can profoundly impact the health and lives of those with sickle cell disease.

Key Points: 
  • We are confident that the programs proposed by this years grantees can profoundly impact the health and lives of those with sickle cell disease.
  • As both a physician who cares for people with sickle cell disease and someone who lives with the disease, I know firsthand the significant impact the organizations awarded a GBT ACCEL grant can make.
  • I thank all the applicants for their innovative proposals to improve SCD care and look forward to following the progress of these inspiring grantees.
  • Founded in 2011, GBT is delivering on its goal to transform the treatment and care of sickle cell disease (SCD), a lifelong, devastating inherited blood disorder.

Newborn Screening Test for Sickle Cell Disease Now Supported by Gazelle 8-Minute, Point-of-Care Diagnostic

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星期一, 六月 14, 2021
Health, Consumer, Technology, Other Health, Mobile, Wireless, Baby, Maternity, Hardware, Medical specialties, Branches of biology, Clinical medicine, Hemoglobins, Health in Africa, Blood disorders, Hematopathology, Sickle cell disease, Respiratory physiology, Hematologic disease, Hemoglobinopathy, Scd

The upgrade, free to those with the Gazelle device, also includes printing enhancements and a variety of ease-of-use features.

Key Points: 
  • The upgrade, free to those with the Gazelle device, also includes printing enhancements and a variety of ease-of-use features.
  • Sickle cell disease (SCD) is one of the most common and severe of all inherited hematological disorders, causing substantial morbidity and mortality worldwide.
  • According to the Sickle Cell Disease Foundation, SCD is responsible for between 5% and 16% of all infant deaths in sub-Saharan Africa.
  • Hemex improvements enable the Gazelle test to identify these low levels for sickle cell disease and trait, Barts, and other important hemoglobinopathies at birth, continued Dr. Obaro.

GBT Announces New Employment Inducement Grants

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星期五, 六月 4, 2021
Health in Africa, Hematopathology, Sickle cell disease, Stearoyl-CoA desaturase-1, Scd, Clinical medicine, Branches of biology, Medical specialties

These awards were made under GBTs Amended and Restated 2017 Inducement Equity Plan (the Plan).

Key Points: 
  • These awards were made under GBTs Amended and Restated 2017 Inducement Equity Plan (the Plan).
  • The above-described awards were each granted as an inducement material to the employees entering into employment with the company in accordance with NASDAQ Listing Rule 5635(c)(4), and were granted pursuant to the terms of the Plan.
  • The Plan was adopted by GBTs board of directors in January 2017 and has been amended and restated from time to time.
  • Founded in 2011, GBT is delivering on its goal to transform the treatment and care of sickle cell disease (SCD), a lifelong, devastating inherited blood disorder.

Aruvant Announces Oral Presentation at 15th Annual Sickle Cell Disease Research & Educational Symposium & 44th National Sickle Cell Disease Scientific Meeting

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星期四, 五月 27, 2021
Branches of biology, Life sciences, Health sciences, Health in Africa, Hematopathology, Sickle cell disease, Gene therapy, Therapy, Scd

NEW YORK and BASEL, Switzerland, May 27, 2021 /PRNewswire/ --Aruvant Sciences ("Aruvant"), a private company focused on developing gene therapies for rare diseases, today announced that data on ARU-1801, a potentially curative lentiviral gene therapy for sickle cell disease (SCD), will be delivered in an oral presentation at the 15th Annual Sickle Cell Disease Research & Educational Symposium & 44th National Sickle Cell Disease Scientific Meeting.

Key Points: 
  • NEW YORK and BASEL, Switzerland, May 27, 2021 /PRNewswire/ --Aruvant Sciences ("Aruvant"), a private company focused on developing gene therapies for rare diseases, today announced that data on ARU-1801, a potentially curative lentiviral gene therapy for sickle cell disease (SCD), will be delivered in an oral presentation at the 15th Annual Sickle Cell Disease Research & Educational Symposium & 44th National Sickle Cell Disease Scientific Meeting.
  • These results indicate that it may be possible to successfully treat individuals with SCD using only reduced intensity conditioning with ARU-1801."
  • Preliminary clinical data from the MOMENTUM study, an ongoing Phase 1/2 trial of ARU-1801 in patients with severe sickle cell disease, demonstrate continuing durable reductions in disease burden.
  • Aruvant is conducting the MOMENTUM study, which is evaluating ARU-1801, a one-time potentially curative investigational gene therapy for patients with SCD.