Degenerative disease

Results from Italfarmaco Pivotal Phase 3 EPIDYS Study of Givinostat in Duchenne Muscular Dystrophy (DMD) Published in The Lancet Neurology

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화요일, 3월 19, 2024

Italfarmaco S.p.A. announced today that the full results from the Company’s pivotal phase 3 EPIDYS clinical trial with givinostat in ambulant boys 6 years of age and older with Duchenne muscular dystrophy (DMD) have been published in The Lancet Neurology.

Key Points: 
  • Italfarmaco S.p.A. announced today that the full results from the Company’s pivotal phase 3 EPIDYS clinical trial with givinostat in ambulant boys 6 years of age and older with Duchenne muscular dystrophy (DMD) have been published in The Lancet Neurology.
  • The full manuscript, titled, “Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomized, double-blind, placebo-controlled, phase 3 trial,” is published on The Lancet Neurology website .
  • Participants attended study site visits every 12 weeks for 72 weeks and were evaluated for all endpoints within the study protocol.
  • Upon completion of the 18-month double-blind period, EPIDYS study participants were eligible to enroll in an open-label extension study (OLE study) to receive givinostat on an ongoing basis.

ZKR Orthopedics Receives IDE Approval for Clinical Trial

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수요일, 2월 14, 2024

SAN FRANCISCO, Feb. 14, 2024 (GLOBE NEWSWIRE) -- ZKR Orthopedics, Inc., a clinical stage medical device company, announced that it has received Investigational Device Exemption (IDE) approval from the United States Food and Drug Administration (FDA) for a multicenter prospective clinical trial.

Key Points: 
  • SAN FRANCISCO, Feb. 14, 2024 (GLOBE NEWSWIRE) -- ZKR Orthopedics, Inc., a clinical stage medical device company, announced that it has received Investigational Device Exemption (IDE) approval from the United States Food and Drug Administration (FDA) for a multicenter prospective clinical trial.
  • We expect the data collected by this trial will establish a strong foundation of compelling clinical evidence for the LIFT implant approval for clinical use," commented David Cash, CEO of ZKR Orthopedics.
  • The IDE approval announcement was made as part of the ZKR Orthopedics activities at the 2024 American Academy of Orthopedic Surgeons annual meeting in San Francisco.
  • In addition to reporting IDE trial approval, the Company participated in the Canaccord Genuity Musculoskeletal Conference earlier this week.

No Decline in Cognition Scores in Patients with Mild Alzheimer's Disease Who Received Simufilam Continuously For 24 Months

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수요일, 2월 7, 2024

ADAS-Cog Scores Were Stable in a Group of Patients with Mild Alzheimer’s Who Received Drug Candidate Simufilam Continuously, Baseline to Month 24.

Key Points: 
  • ADAS-Cog Scores Were Stable in a Group of Patients with Mild Alzheimer’s Who Received Drug Candidate Simufilam Continuously, Baseline to Month 24.
  • Mild Alzheimer’s Patients Who Received Simufilam Non-Continuously Declined a Group Average of 1 Point on ADAS-Cog, Baseline to Month 24.
  • Average changes in ADAS-Cog scores, baseline to month 24, indicate the following:
    Patients with mild Alzheimer’s disease who received simufilam treatment continuously for two years (n=47) had no decline in ADAS-Cog scores (± 1.51 SE) as a group.
  • Patients with mild Alzheimer’s who received simufilam treatment non-continuously (n=40) declined 1 point on ADAS-Cog (± 1.65 SE) as a group.

Ophthalmology innovations paving the way for diagnosis & treatment of age-related macular degeneration (AMD)

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수요일, 2월 7, 2024

TORONTO, Feb. 7, 2024 /CNW/ - Age-related macular degeneration (AMD) is one of the leading causes of vision loss for people over the age of 55 and today, affecting approximately 2.5 million Canadians.

Key Points: 
  • TORONTO, Feb. 7, 2024 /CNW/ - Age-related macular degeneration (AMD) is one of the leading causes of vision loss for people over the age of 55 and today, affecting approximately 2.5 million Canadians.
  • As February marks AMD Awareness Month, the Canadian Ophthalmological Society (COS) is educating Canadians about the importance of early diagnosis and treatment, along with vision care innovations that will play a key role in slowing down the progression of this eye disease.
  • AMD is a degenerative disease that happens when part of the retina called the macula is damaged.
  • To learn more about AMD, its risk factors, diagnosis and treatment visit www.see the possibilities.ca .

Novel AI platform matches cardiologists in detecting rheumatic heart disease

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화요일, 1월 16, 2024

RHD is caused by the body’s reaction to repeated Strep A bacterial infections and can cause permanent heart damage.

Key Points: 
  • RHD is caused by the body’s reaction to repeated Strep A bacterial infections and can cause permanent heart damage.
  • This tell-tale sign of the disease causes the mitral valve flaps to close improperly, leading to backward blood flow in the heart.
  • They combined the power of both approaches to optimize the novel algorithm, which is trained to interpret ultrasound images of the heart to detect RHD.
  • Already, the AI algorithm has analyzed 39 features of hearts with RHD that cardiologists cannot detect or measure with the naked eye.

AI-Powered Avatar from Lenovo, DeepBrain AI, and the Scott-Morgan Foundation Creates New Communication Possibilities for People with Severe Disabilities

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화요일, 1월 9, 2024

As part of an ongoing collaboration with the Scott-Morgan Foundation , Lenovo proposed developing an AI-powered avatar to leap past traditional voice synthesis and transform communication and connection for people with severe disabilities.

Key Points: 
  • As part of an ongoing collaboration with the Scott-Morgan Foundation , Lenovo proposed developing an AI-powered avatar to leap past traditional voice synthesis and transform communication and connection for people with severe disabilities.
  • Lenovo then approached DeepBrain AI , a leader in next-gen avatars and a Lenovo AI Innovator partner , to use its generative AI technology.
  • Sponsored by Lenovo, the DeepBrain AI team captured a detailed full-body video of Taylor in its California studio.
  • The video became the foundational data for her new AI avatar, debuted at CES 2024, and offering 96 percent true-to-life accuracy.

VIVUS Provides Update on Pipeline and Program Milestones

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월요일, 1월 8, 2024

CAMPBELL, Calif., Jan. 08, 2024 (GLOBE NEWSWIRE) -- VIVUS LLC, a biopharmaceutical company committed to the development and commercialization of innovative therapies that focus on advancing treatments for patients with serious unmet medical needs, today announced pipeline updates and program milestones.

Key Points: 
  • CAMPBELL, Calif., Jan. 08, 2024 (GLOBE NEWSWIRE) -- VIVUS LLC, a biopharmaceutical company committed to the development and commercialization of innovative therapies that focus on advancing treatments for patients with serious unmet medical needs, today announced pipeline updates and program milestones.
  • “VIVUS has been continuously dedicated to addressing the therapeutic needs of patients with serious medical conditions and life-limiting diseases, including exocrine pancreatic insufficiency, obesity and pulmonary arterial hypertension,” said John Amos, Chief Executive Officer at VIVUS LLC.
  • QSYMIA® is now the number one branded oral product in the US for obesity treatment.
  • Moreover, VIVUS is actively developing pipeline products focusing on bone marrow transplant preparation (VI-0609) and diabetes treatments (VI-0809 and VI-0810).”

MitoRx Therapeutics Announces Close of Seed Extension Financing Round to Advance its Mitochondrial-Protective Therapeutics to Treat Degenerative Diseases

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수요일, 12월 13, 2023

OXFORD, United Kingdom, Dec. 13, 2023 (GLOBE NEWSWIRE) -- MitoRx Therapeutics (MitoRx), a biotechnology company developing novel therapeutics that reverse mitochondrial dysfunction to arrest the progression of degenerative diseases, announces the final close of its seed extension financing round, with £4m raised to date from existing investors.

Key Points: 
  • OXFORD, United Kingdom, Dec. 13, 2023 (GLOBE NEWSWIRE) -- MitoRx Therapeutics (MitoRx), a biotechnology company developing novel therapeutics that reverse mitochondrial dysfunction to arrest the progression of degenerative diseases, announces the final close of its seed extension financing round, with £4m raised to date from existing investors.
  • The platform is extendable to other degenerative diseases, and additional evidence has been generated in a mammalian respiratory disease model through its academic collaborators.
  • Dr. Jon Rees, Chief Executive Officer of MitoRx, said, “Degenerative diseases place great burdens on patients and healthcare systems.
  • This seed extension financing round enables us to advance a lead asset toward the clinic and provides a firm foundation to secure a Series A to take us to the clinic within two years.

Charles River and Genetic Cures for Kids Announce Gene Therapy Manufacturing Collaboration

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화요일, 11월 28, 2023

Charles River Laboratories International, Inc. (NYSE: CRL) and Genetic Cures for Kids Inc (GC4K), an Australian non-profit foundation focused on research programs and clinical trials to find cures for rare diseases, today announced a plasmid DNA contract development and manufacturing organization (CDMO) collaboration.

Key Points: 
  • Charles River Laboratories International, Inc. (NYSE: CRL) and Genetic Cures for Kids Inc (GC4K), an Australian non-profit foundation focused on research programs and clinical trials to find cures for rare diseases, today announced a plasmid DNA contract development and manufacturing organization (CDMO) collaboration.
  • In recent years, Charles River has significantly broadened its cell and gene therapy portfolio to simplify complex supply chains and meet growing demand for plasmid DNA, viral vector, and cell therapy services.
  • Combined with the Company’s legacy testing capabilities, Charles River offers a comprehensive “concept-to-cure” advanced therapies solution.
  • - Golden Whitrod, Tallulah Moon’s mother and Co-Founder and President, Genetic Cures for Kids, Inc.

Art Blocks to Host Auction of Generative Art to Benefit Cure Alzheimer's Fund

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금요일, 10월 20, 2023

Proceeds from the auction of 1,000 NFTs (200 per day) will be donated to Cure Alzheimer's Fund.

Key Points: 
  • Proceeds from the auction of 1,000 NFTs (200 per day) will be donated to Cure Alzheimer's Fund.
  • The upcoming auction supports the work of Cure Alzheimer's Fund, a non-profit organization dedicated to funding research with the highest probability of preventing, slowing or reversing Alzheimer's disease.
  • A week-long charitable event featuring daily auctions of 200 works by five leading generative artists, minted on Art Blocks.
  • Art Blocks , a platform dedicated to bringing compelling works of contemporary generative art to life.