Priapism

Erections: what we learned from mice could help with sexual health in men

Retrieved on: 
星期五, 二月 9, 2024

Using a technique to render cells light-sensitive enabled us to activate fibroblasts by shining blue light from outside onto the penises of mice.

Key Points: 
  • Using a technique to render cells light-sensitive enabled us to activate fibroblasts by shining blue light from outside onto the penises of mice.
  • More fibroblasts, more blood flow
    Surprisingly, we found that the number of penile fibroblasts changed in relation to the frequency of erections.
  • Studying penises of aged mice, we found that they possessed a lower number of fibroblasts compared with young mice.
  • Our research unveils a mechanism for controlling penile erections, opening the door for further exploration to understand and improve sexual health.

Erections: what we learned from mice could help with sexual health in humans

Retrieved on: 
星期四, 二月 8, 2024

Using a technique to render cells light-sensitive enabled us to activate fibroblasts by shining blue light from outside onto the penises of mice.

Key Points: 
  • Using a technique to render cells light-sensitive enabled us to activate fibroblasts by shining blue light from outside onto the penises of mice.
  • More fibroblasts, more blood flow
    Surprisingly, we found that the number of penile fibroblasts changed in relation to the frequency of erections.
  • Studying penises of aged mice, we found that they possessed a lower number of fibroblasts compared with young mice.
  • Our research unveils a mechanism for controlling penile erections, opening the door for further exploration to understand and improve sexual health.

Long-term Follow-up Data From bluebird’s Gene Therapy Program in Sickle Cell Disease Support Durable, Potentially Curative Benefits Through Stable Production of Anti-Sickling Adult Hemoglobin and Resolution of Vaso-Occlusive Events

Retrieved on: 
星期六, 十二月 9, 2023

The majority of adverse events in treated patients were attributed to underlying sickle cell disease or conditioning with busulfan.

Key Points: 
  • The majority of adverse events in treated patients were attributed to underlying sickle cell disease or conditioning with busulfan.
  • Lovo-cel is the most deeply studied gene therapy in development for sickle cell disease, with the most patients treated and longest follow-up in the field.
  • As of February 13, 2023, 59 patients were treated across the entire clinical development program with follow-up beyond 8 years in the earliest treated patients.
  • In sickle cell disease, hemolysis happens too quickly due to the fragility of sickled red blood cells, resulting in hemolytic anemia.

bluebird bio Announces FDA Approval of LYFGENIA™ (lovotibeglogene autotemcel) for Patients Ages 12 and Older with Sickle Cell Disease and a History of Vaso-Occlusive Events

Retrieved on: 
星期五, 十二月 8, 2023

LYFGENIA is a one-time gene therapy that has the potential to resolve vaso-occlusive events and is custom-designed to treat the underlying cause of sickle cell disease.

Key Points: 
  • LYFGENIA is a one-time gene therapy that has the potential to resolve vaso-occlusive events and is custom-designed to treat the underlying cause of sickle cell disease.
  • “LYFGENIA has the potential to have a transformational impact for patients who currently live under the shadow of unpredictable and debilitating vaso-occlusive events.
  • The burden associated with VOEs is pervasive and can affect every aspect of life for patients and their families and caregivers.
  • A replay of the webcast will be available on the bluebird website for 90 days following the event.

bluebird bio Details Plans for the Commercial Launch of LYFGENIA™ Gene Therapy for Patients Ages 12 and Older with Sickle Cell Disease and a History of Vaso-Occlusive Events

Retrieved on: 
星期五, 十二月 8, 2023

“Our approach is built on the principle that timely, equitable access to gene therapy is imperative for people living with sickle cell disease,” said Tom Klima, chief commercial & operating officer, bluebird bio.

Key Points: 
  • “Our approach is built on the principle that timely, equitable access to gene therapy is imperative for people living with sickle cell disease,” said Tom Klima, chief commercial & operating officer, bluebird bio.
  • The burden of sickle cell disease is immense and can impact every aspect of life for patients and their loved ones.
  • Fifty to 60% of adults living with sickle cell disease have end organ damage, and one in four people living with sickle cell disease have a stroke by age 45.
  • “The FDA approval of a sickle cell disease gene therapy may be a game changer,” said Dr. Shantel Herbert-Magee, Chief Medical Director, Louisiana Medicaid.

Three Animations from Elara Systems Won Awards at the 2023 AMI Conference

Retrieved on: 
星期一, 八月 7, 2023

GOLD RIVER, Calif., Aug. 7, 2023 /PRNewswire-PRWeb/ -- The Group's "Sickle Cell Associated Priapism in Children" Video Received the Member's Choice Award

Key Points: 
  • In July, three medical animations by Elara Systems won awards at the 77th Annual Association of Medical Illustrators (AMI) Conference in Henderson, Nevada.
  • In July, three medical animations by Elara Systems won awards at the 77th Annual Association of Medical Illustrators (AMI) Conference in Henderson, Nevada.
  • Elara Systems is honored to have received these awards from members of the AMI community.
  • The company currently employs medical animators who serve as active AMI members to ensure its projects meet the highest standards in medical visualization.

Rhythm Pharmaceuticals Announces New Employment Inducement Grants

Retrieved on: 
星期三, 十二月 7, 2022

About Rhythm PharmaceuticalsRhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases. Rhythm’s precision medicine, setmelanotide, is approved by the U.S. Food and Drug Administration (FDA) for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency confirmed by genetic testing, or patients with a clinical diagnosis of Bardet-Biedl syndrome (BBS). The European Commission (EC) and Great Britain’s Medicines & Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare genetic diseases of obesity and is leveraging the Rhythm Engine and the largest known obesity DNA database -- now with approximately 45,000 sequencing samples -- to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. Rhythm’s headquarters is in Boston, MA.

Key Points: 

    Rhythm Pharmaceuticals to Present at BofA Securities 2022 Virtual Biotech SMID Cap Conference

    Retrieved on: 
    星期四, 十二月 1, 2022

    About Rhythm PharmaceuticalsRhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases. Rhythm’s precision medicine, setmelanotide, is approved by the U.S. Food and Drug Administration (FDA) for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency confirmed by genetic testing, or patients with a clinical diagnosis of Bardet-Biedl syndrome (BBS). The European Commission (EC) and Great Britain’s Medicines & Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare genetic diseases of obesity and is leveraging the Rhythm Engine and the largest known obesity DNA database -- now with approximately 45,000 sequencing samples -- to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. Rhythm’s headquarters is in Boston, MA.

    Key Points: 
    • A replay of the webcast will be available on the Rhythm website for 30 days following the presentation.
    • 2022 Rhythm Pharmaceuticals, Inc. All Rights Reserved.
    • Inthe United StatesandEurope, setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology.
    • This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995.

    Rhythm Pharmaceuticals Announces New Employment Inducement Grants

    Retrieved on: 
    星期一, 十一月 7, 2022

    About Rhythm PharmaceuticalsRhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases. Rhythm’s precision medicine, setmelanotide, is approved by the U.S. Food and Drug Administration (FDA) for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency confirmed by genetic testing, or patients with a clinical diagnosis of Bardet-Biedl syndrome (BBS). The European Commission (EC) has authorized setmelanotide for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above.The UK’s Medicines & Healthcare Products Regulatory Agency (MHRA) authorized setmelanotide for the treatment of obesity and the control of hunger associated with genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare genetic diseases of obesity and is leveraging the Rhythm Engine and the largest known obesity DNA database -- now with approximately 45,000 sequencing samples -- to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. Rhythm’s headquarters is in Boston, MA.

    Key Points: 
    • These inducement stock options and inducement RSUs are subject to the terms of the Rhythm Pharmaceuticals, Inc. 2022 Employment Inducement Plan (the "Inducement Plan").
    • The Inducement Plan is used exclusively for the grant of equity awards to individuals as an inducement material to the employees entering into employment with Rhythm pursuant to Nasdaq Listing Rule 5635(c)(4).
    • The Inducement Plan was adopted by Rhythms board of directors on February 9, 2022.
    • Rhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases.

    Tris Pharma Announces US District Court Upholds Validity of QuilliChew ER® Patents; Rules Teva Infringes Tris Patents

    Retrieved on: 
    星期四, 八月 18, 2022

    The ruling prevents Teva from launching generic versions of QuilliChew ER until the expiration of Tris's patent portfolio providing exclusivity until August 2033.

    Key Points: 
    • The ruling prevents Teva from launching generic versions of QuilliChew ER until the expiration of Tris's patent portfolio providing exclusivity until August 2033.
    • "The decision of the District Court not only confirms the validity and infringement of our QuilliChew ER patents, but also validates the innovative nature of our LiquiXRtechnology which we utilized to develop QuilliChew ER and our other branded ADHD medications."
    • QuilliChew ER is approved for treatment of ADHD in people 6 years and older.
    • Call the doctor right away if any signs of unexplained wounds appear on fingers or toes while taking QuilliChew ER.