Cure Rare Disease

Muscular Dystrophy Association Announces Seven Recipient Organizations of Advocacy Collaboration Grants

Retrieved on: 
Thursday, February 1, 2024
Policy, Occupancy, CFO, Hospital, Neuromuscular disease, Travel, Wheelchair, MDA, Therapy, Muscular Dystrophy Association, Muscular dystrophy, Caregiver, Child, Safety, Patient, CMD, Atmosphere, Cure Rare Disease, Disease, Public policy, Air travel, NMD, VCP, DMD, Empowerment, Organization, Nursing

“Muscular Dystrophy Association is proud to announce the recipients of the MDA Advocacy Collaboration Grants.

Key Points: 
  • “Muscular Dystrophy Association is proud to announce the recipients of the MDA Advocacy Collaboration Grants.
  • “The Child Neurology Foundation is thrilled to be a recipient of Muscular Dystrophy Association’s Advocacy Collaboration Grants program to support the development of a comprehensive Needs Assessment Survey,” said Katie Hentges, Director of Programs.
  • “The Muscular Dystrophy Association Advocacy Collaboration Grant will enable Cure CMD to continue its legislative advocacy work and provide skills training, resources, and advocacy opportunities for the CMD community,” said Lani Knutson, Cure CMD Advocacy Team Lead.
  • Other innovative programs, campaigns, or initiatives that can measurably impact one or more of MDA and the collaborating organizations’ common advocacy goals.

Cure Rare Disease Receives Muscular Dystrophy Association Grant

Retrieved on: 
Thursday, February 16, 2023
Health, Genetics, Other Health, Clinical Trials, General Health, Biotechnology, Organization, Cure Rare Disease, ALS, Muscular Dystrophy Association, Therapy, MDA, Rare, Drug development, Neuromuscular disease, Patient, Muscular dystrophy, Ecosystem, Food, Research, Rare disease, Disease, Food and Drug Administration Amendments Act of 2007, Public policy, Pharmaceutical industry

Cure Rare Disease , a clinical-stage nonprofit biotechnology company, has been awarded an advocacy collaboration grant from the Muscular Dystrophy Association (MDA) to support research on novel reimbursement strategies for drugs developed for ultra-rare diseases, a key challenge for rare disease patients, including muscular dystrophy patients.

Key Points: 
  • Cure Rare Disease , a clinical-stage nonprofit biotechnology company, has been awarded an advocacy collaboration grant from the Muscular Dystrophy Association (MDA) to support research on novel reimbursement strategies for drugs developed for ultra-rare diseases, a key challenge for rare disease patients, including muscular dystrophy patients.
  • “We are honored to have MDA's support as we look to tackle one of the greatest barriers for ultra-rare disease patients to access potentially life-saving medicines,” said Rich Horgan, founder and chief executive officer of Cure Rare Disease.
  • Our goal is to find a solution to this critical economic challenge.”
    A leading health organization in the United States for people living with muscular dystrophy, ALS and related neuromuscular diseases, the Muscular Dystrophy Association has awarded the grant to Cure Rare Disease as part of the organization’s Advocacy Collaboration Grants Program, which seeks to fund public policy and advocacy projects that will benefit the neuromuscular disease community.
  • Cure Rare Disease considers the challenge of reimbursement for ultra-rare diseases to be closely tied to its mission of developing 18 potential therapeutics in its pipeline of rare, neuromuscular conditions.

Cure Rare Disease Acquires Laboratory Space for New Headquarters in Connecticut

Retrieved on: 
Thursday, September 29, 2022
Research, Commercial Building & Real Estate, Construction & Property, Genetics, Philanthropy, Other Health, Biotechnology, Fund Raising, Health, Science, Therapy, HRSA, D, Research, Health Resources and Services Administration, Rare disease, Organization, Growth, Degenerative disease, Patient, List of life sciences, CEO, CRD, Cure Rare Disease, CT, Ecosystem, Biotechnology, Medicine, Multimedia, BD, Disease, Pharmaceutical industry

Cure Rare Disease (CRD), a clinical-stage nonprofit biotechnology company, has acquired its first laboratory space at 4 Research Drive in Woodbridge, Connecticut, where the organization will relocate its headquarters and expand its research and development operations.

Key Points: 
  • Cure Rare Disease (CRD), a clinical-stage nonprofit biotechnology company, has acquired its first laboratory space at 4 Research Drive in Woodbridge, Connecticut, where the organization will relocate its headquarters and expand its research and development operations.
  • CRD will also make 15,000 square feet of space available for lease to other life science and healthcare firms.
  • "AdvanceCT is thrilled to welcome Cure Rare Disease to Connecticut, said Tim Miller, Director of BD, Life Sciences, at AdvanceCT.
  • Cure Rare Disease, a 501(c)(3) nonprofit biotechnology company based in Boston, is transforming possibilities for people with rare diseases by developing advanced therapeutics in time to save lives.

Global Genes Honors a Diverse Group of International Rare Disease Leaders at the 2022 RARE Champions of Hope Celebration

Retrieved on: 
Wednesday, September 14, 2022
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Global Genes , a leading rare disease patient advocacy organization, today announced its annual RARE Champions of Hope award winners, recognizing leaders and organizations for their inspiring work in driving innovation, advocacy and change in rare disease as part of the 2022 RARE Disease Patient Advocacy Summit in San Diego.

Key Points: 
  • Global Genes , a leading rare disease patient advocacy organization, today announced its annual RARE Champions of Hope award winners, recognizing leaders and organizations for their inspiring work in driving innovation, advocacy and change in rare disease as part of the 2022 RARE Disease Patient Advocacy Summit in San Diego.
  • This year, the celebration recognizes those who made a significant impact across seven categories in the areas of advocacy, industry, medical care, and science, as well as up-and-coming rare disease leaders.
  • Global Genes is a 501(c)(3) non-profit organization dedicated to creating a globally connected community committed to eliminating the challenges of rare disease.
  • If you or someone you love has a rare disease or are searching for a diagnosis, contact Global Genes at 949-248-RARE or visit our Resource Hub.

Cure Rare Disease Receives FDA Approval to Administer First-in-Human CRISPR Therapeutic

Retrieved on: 
Wednesday, August 10, 2022
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Cure Rare Disease (CRD) - a Boston-based 501c3 nonprofit biotech - announces the approval from the U.S. Food and Drug Administration (FDA) to administer its very first therapeutic.

Key Points: 
  • Cure Rare Disease (CRD) - a Boston-based 501c3 nonprofit biotech - announces the approval from the U.S. Food and Drug Administration (FDA) to administer its very first therapeutic.
  • "It is inspiring to see Cure Rare Disease achieve FDA approval for the first-in-human IND using CRISPR-transactivator technology to treat a rare mutation causing Duchenne muscular dystrophy.
  • As a 501c3 nonprofit biotechnology organization, the therapeutics developed by Cure Rare Disease and its collaborators are largely funded with support from its grassroots community of rare disease patients, families, and corporate partners.
  • Cure Rare Disease, a 501(c)(3) nonprofit biotechnology company based in Boston, is transforming possibilities for people with rare diseases by developing advanced therapeutics in time to save lives.

VectorY Raises €31 Million in Seed Financing to Develop Next-Generation Gene Therapies Aimed at Muscular and CNS Disorders

Retrieved on: 
Tuesday, June 15, 2021
Biotechnology, Pharmaceutical, Health, Clinical trials, Branches of biology, Biology, Life sciences, Biotechnology, Applied genetics, Molecular biology, Gene therapy, Cure Rare Disease

VectorY Therapeutics, a biotech company focusing on the development of innovative gene therapy approaches for the treatment of muscular and neurodegenerative disorders through vectorized antibodies, today announces the completion of a 31 million seed financing round.

Key Points: 
  • VectorY Therapeutics, a biotech company focusing on the development of innovative gene therapy approaches for the treatment of muscular and neurodegenerative disorders through vectorized antibodies, today announces the completion of a 31 million seed financing round.
  • VectorY was launched in October 2020, and has established its laboratories and offices at the Amsterdam Science Park in The Netherlands.
  • The Company is developing a pipeline of vectorized antibodies targeting muscular and CNS diseases, based on a novel AAV platform and innovative antibody-based targeted degradation technologies.
  • VectorY combines the therapeutic potential of antibodies, RNA and gene therapy to develop long-lasting therapeutic solutions for muscular and neurodegenerative diseases with high unmet medical need.

CRISPR Therapeutics Provides Business Update and Reports First Quarter 2021 Financial Results

Retrieved on: 
Tuesday, April 27, 2021
Branches of biology, Biology, Life sciences, Biotechnology, Genetic engineering, Genome editing, CRISPR Therapeutics, CRISPR, American Association for Cancer Research, Cure Rare Disease, Intellia Therapeutics, Branches of biology, Biology, Life sciences, Biotechnology, Genetic engineering, Genome editing, CRISPR Therapeutics, CRISPR, American Association for Cancer Research, Cure Rare Disease, Intellia Therapeutics

Earlier this month, the Company announced preclinical data from its CAR-T program at the American Association for Cancer Research (AACR) Annual Meeting 2021.

Key Points: 
  • Earlier this month, the Company announced preclinical data from its CAR-T program at the American Association for Cancer Research (AACR) Annual Meeting 2021.
  • CRISPR Therapeutics will continue to support the development of CTX001 and invest in further innovation to maximize its potential.
  • Under a recently amended collaboration agreement, Vertex will lead global development and commercialization of CTX001 and split program costs and profits worldwide 60/40 with CRISPR Therapeutics.
  • CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases.

Global Genes, Leaders in Life Sciences and Pharma, and Informa’s Biotech Showcase™ Partner to Connect Rare Disease Advocates, Investors and Companies at RARE Beyond the Square

Retrieved on: 
Thursday, December 17, 2020
Health, Genetics, General Health, Research, Science, Pharmaceutical, Biotechnology, Articles, Biotechnology, Medicine, Cure Rare Disease, Life sciences, Rare disease, Global Genes, UCB, Disease

Global Genes , a leading rare disease patient advocacy organization, announced a partnership with the organizers of the Biotech Showcase to virtually host RARE Beyond the Square Jan. 11-14, 2021, to highlight rare disease innovation, share information, and facilitate partnering and networking among companies, investors, and rare disease communities.

Key Points: 
  • Global Genes , a leading rare disease patient advocacy organization, announced a partnership with the organizers of the Biotech Showcase to virtually host RARE Beyond the Square Jan. 11-14, 2021, to highlight rare disease innovation, share information, and facilitate partnering and networking among companies, investors, and rare disease communities.
  • RARE Beyond the Square plants a flag for rare disease in the midst of a seminal gathering of global leaders that sets trends and investment priorities in healthcare for the year.
  • All rare disease patients, advocates, and partners are welcome to attend this years event virtually from Jan. 11-14, 2021.
  • RARE Beyond the Square is supported by bluebird bio, Charles River Laboratories, Cure Rare Disease, Deerfield Management, Sarepta Therapeutics, UCB, and Vertex Pharmaceuticals.