Inclusion bodies

FirstEnergy Honored by Women's Business Enterprise Center for Supplier Diversity Programs

Retrieved on:

Thursday, September 23, 2021

FirstEnergy's Supplier Diversity Program provides opportunities in a competitive environment to small, woman-owned; minority-owned; HUBZone; veteran-owned; and service-disabled veteran-owned businesses.

Key Points:

FirstEnergy's Supplier Diversity Program provides opportunities in a competitive environment to small, woman-owned; minority-owned; HUBZone; veteran-owned; and service-disabled veteran-owned businesses.
It serves as just one part of the company's commitment to diversity, equity and inclusion, a FirstEnergy core value.
In 2020, FirstEnergy spent $482 million with diverse suppliers, or about 16% of the company's total supplier expenditures.
The award is the latest recognition of FirstEnergy's leading diversity and inclusion and supplier diversity programs.

CytRx Notes Orphazyme’s Announcement of Topline Results from Trial for Arimoclomol in the Treatment of Inclusion Body Myositis

Retrieved on:

Thursday, April 1, 2021

According to Orphazyme, the primary goal was to evaluate the treatment effect on disease progression as measured by the inclusion body myositis functional rating scale.

Key Points:

According to Orphazyme, the primary goal was to evaluate the treatment effect on disease progression as measured by the inclusion body myositis functional rating scale.
Participants were randomized (1:1 ratio) to receive either arimoclomol citrate (400 mg three times daily) or placebo for up to 20 months.
Orphazyme is developing arimoclomol in four indications including Amyotrophic Lateral Sclerosis ("ALS"), Niemann-Pick disease Type C ("NPC"), Gaucher disease and Inclusion Body Myositis ("IBM").
Arimoclomol, the companys lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C, Gaucher disease, Inclusion Body Myositis, and Amyotrophic Lateral Sclerosis.

Orphazyme announces topline results from pivotal trial of arimoclomol for Inclusion Body Myositis (IBM)

Retrieved on:

Monday, March 29, 2021

Branches of biology, Neurological disorders, Biology, Rare diseases, Neuroscience, Amyotrophic lateral sclerosis, Inclusion bodies, Myositis, Neurodegeneration, Mecasermin rinfabate

The primary goal was to evaluate the treatment effect on disease progression as measured by the inclusion body myositis functional rating scale (IBMFRS).

Key Points:

The primary goal was to evaluate the treatment effect on disease progression as measured by the inclusion body myositis functional rating scale (IBMFRS).
Participants were randomized (1:1 ratio) to receive either arimoclomol citrate (400 mg three times daily) or placebo for up to 20 months.
We recognize these data are disappointing for patients and families who continue to eagerly await a promising option for IBM.
Orphazyme expects data from a pivotal Phase 3 trial of arimoclomol in Amyotrophic Lateral Sclerosis (ALS), a neurodegenerative disease, this spring.

CytRx Highlights Orphazyme’s Momentum Ahead of Potential Regulatory Approval of Arimoclomol for Niemann-Pick Disease Type C

Retrieved on:

Monday, March 8, 2021

Oncology, FDA, Health, Clinical trials, Pharmaceutical, Biotechnology, Rare diseases, Neurological disorders, Branches of biology, Lipid storage disorders, Clinical medicine, Inclusion bodies, Niemann–Pick disease, type C, Myositis, Arimoclomol, Niemann, Amyotrophic lateral sclerosis, Amyotrophy

CytRx has an agreement with Orphazyme that can yield potential milestone payments and future single and double-digit royalties paid on sales of arimoclomol.

Key Points:

CytRx has an agreement with Orphazyme that can yield potential milestone payments and future single and double-digit royalties paid on sales of arimoclomol.
Orphazyme now expects to receive a decision on European regulatory approval for arimoclomol for NPC by Q4 2021.
Orphazyme is developing arimoclomol in four indications including Amyotrophic Lateral Sclerosis ("ALS"), Niemann-Pick disease Type C ("NPC"), Gaucher disease and Inclusion Body Myositis ("IBM").
Arimoclomol, the companys lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C, Gaucher disease, Inclusion Body Myositis, and Amyotrophic Lateral Sclerosis.

Orphazyme to showcase data on arimoclomol in Niemann-Pick disease Type C during the 2021 Annual WORLDSymposium™

Retrieved on:

Thursday, February 4, 2021

Rare diseases, Lipid storage disorders, Branches of biology, Clinical medicine, Health, Niemann–Pick disease, type C, Niemann–Pick disease, Niemann, Type C, Inclusion bodies, NPC, Gaucher's disease

We look forward to convening with the research community during the conference and to showcasing these data in support of arimoclomol.

Key Points:

We look forward to convening with the research community during the conference and to showcasing these data in support of arimoclomol.
We continue to engage in productive dialogue with the FDA related to the NDA for arimoclomol, said Thomas Blaettler, Chief Medical Officer, Orphazyme.
Arimoclomol, the companys lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C (NPC), Amyotrophic Lateral Sclerosis (ALS), Inclusion Body Myositis (IBM) and Gaucher disease.
Niemann-Pick disease Type C (NPC) is a rare, genetic, progressively debilitating, and often fatal neurovisceral disease.

CytRx Issues Statement Regarding U.S. Regulatory Review of Arimoclomol for Niemann-Pick Disease Type C

Retrieved on:

Thursday, December 31, 2020

Notably, the FDA has confirmed that the extension does not impede eligibility for a Pediatric Rare Disease Priority Review Voucher.

Key Points:

Notably, the FDA has confirmed that the extension does not impede eligibility for a Pediatric Rare Disease Priority Review Voucher.
Arimoclomol previously received the FDAs Fast-Track and Breakthrough Therapy Designations for NPC as well as Orphan Drug and Rare Pediatric Disease Designations.
Orphazyme is developing arimoclomol in four indications including Amyotrophic Lateral Sclerosis (ALS), Niemann-Pick disease Type C (NPC), Gaucher disease and sporadic Inclusion Body Myositis (sIBM).
Arimoclomol, the companys lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C, Gaucher disease, sporadic Inclusion Body Myositis, and Amyotrophic Lateral Sclerosis.

The Myositis Association Announces Chrissy Thornton as Executive Director

Retrieved on:

Thursday, December 17, 2020

Medical specialties, Clinical medicine, Medicine, Myositis, Autoimmune diseases, Dermatomyositis, Polymyositis, Myopathy, Inclusion body myositis, Inclusion bodies

ALEXANDRIA, Va., Dec. 17, 2020 /PRNewswire/ -- Afteran extensive and rigorous search, The Myositis Association (TMA) is thrilled to announce that Chrissy Thornton will be joining the Association as Executive Director.In this position, Chrissy willbe responsible forthe operation of the organization as it fulfills its mission to improve the lives of persons affected by myositis, fund innovative research and increase awareness.

Key Points:

ALEXANDRIA, Va., Dec. 17, 2020 /PRNewswire/ -- Afteran extensive and rigorous search, The Myositis Association (TMA) is thrilled to announce that Chrissy Thornton will be joining the Association as Executive Director.In this position, Chrissy willbe responsible forthe operation of the organization as it fulfills its mission to improve the lives of persons affected by myositis, fund innovative research and increase awareness.
Chrissy has abachelor's degreein Sociology and Criminal Justice, and an MBA in Organizational Management from the University of Phoenix.
There are several different forms of myositis diseases including dermatomyositis, polymyositis, inclusion body myositis, necrotizing myopathy, and juvenile forms of the disease.
The Myositis Association is the leading international nonprofit organization committed to the global community of people living with myositis, their care partners, family members, and the medical community.

CytRx Announces Orphazyme’s U.S. Expansion Ahead of Potential Regulatory Approval of Arimoclomol for Niemann-Pick Disease Type C

Retrieved on:

Monday, December 7, 2020

CytRx has an agreement with Orphazyme that can yield potential milestone payments and future royalties paid on sales of arimoclomol.

Key Points:

CytRx has an agreement with Orphazyme that can yield potential milestone payments and future royalties paid on sales of arimoclomol.
Orphazymes U.S. team, which is focused on regulatory review efforts and a first potential commercial launch in 2021, includes legal, commercial, finance, advocacy relations, and regulatory and medical affairs functions.
Orphazyme is developing arimoclomol in four indications including Amyotrophic Lateral Sclerosis (ALS), Niemann-Pick disease Type C (NPC), Gaucher disease and sporadic Inclusion Body Myositis (sIBM).
Arimoclomol, the companys lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C, Gaucher disease, sporadic Inclusion Body Myositis, and Amyotrophic Lateral Sclerosis.

CytRx Highlights Orphazyme’s Submission of European Marketing Authorisation for Arimoclomol to Treat Niemann-Pick Disease Type C

Retrieved on:

Wednesday, November 11, 2020

CytRx has an agreement with Orphazyme that can yield potential milestone payments and future royalties paid on sales of arimoclomol.

Key Points:

CytRx has an agreement with Orphazyme that can yield potential milestone payments and future royalties paid on sales of arimoclomol.
Given the lack of treatment options for individuals afflicted with NPC, arimoclomol represents a possible breakthrough for patients with this brutal disease.
Orphazyme is developing arimoclomol in four indications including amyotrophic lateral sclerosis (ALS), Niemann-Pick disease Type C (NPC), Gaucher disease and sporadic Inclusion Body Myositis (sIBM).
Arimoclomol, the companys lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C, Gaucher disease, sporadic Inclusion Body Myositis, and Amyotrophic Lateral Sclerosis.

The Myositis Association Takes the Lead in Rare Disease in Virtual Community Building and Empowering the Patient through Telemedicine

Retrieved on:

Tuesday, May 5, 2020

Medical specialties, Medicine, Anatomy, Myositis, Autoimmune diseases, Connective tissue diseases, Dermatomyositis, Polymyositis, Myopathy, Inclusion body myositis, Inclusion bodies

The Myositis Association would like to extend our sincerest gratitude to the sponsors who have made this event possible.

Key Points:

The Myositis Association would like to extend our sincerest gratitude to the sponsors who have made this event possible.
Throughout Myositis Awareness Month, TMA will be launching an awareness campaign including a patient empowerment tool called "My Myositis Tracker."
Myositis has several forms, including dermatomyositis, polymyositis, inclusion body myositis, necrotizing myopathy, and juvenile forms of the disease.
The Myositis Association is the leading international nonprofit organization committed to the global community of people living with myositis, their care partners, family members, and the medical community.