rare disease News
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RareStone won the Golden Snail Award 2022
RareStone announced that RareStone and Biogen are named the winner of the Golden Snail Award 2022. Sponsored by the Chinese Organization for Rare Disorders, the prestigious Golden Snail Award is the first award in China to recognize outstanding contributors to rare diseases. Aiming to improve the lives of patients and families impacted by rare diseases, RareStone is building a "RareDisease ...
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The China Alliance for Rare Diseases (CHARD) and the Chinese Red Cross Foundation (CRCF) jointly initiated the `Rare Disease Mutual Assistance Fund`.
RareStone Group provided the first batch of donations to the foundation. December 18th, during the 2021 China Conference on Rare Diseases in Beijing, the China Alliance for Rare Diseases (CHARD) and the Chinese Red Cross Foundation (CRCF) jointly initiated the "Rare Disease Mutual Assistance Fund." The "Rare Disease Mutual Aid Fund" is dedicated to improving rare disease awareness, ...
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Amicus Therapeutics Announces Second Quarter 2022 Financial Results
PHILADELPHIA, Aug. 04, 2022 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on developing and commercializing novel medicines for rare diseases, today announced financial results for the quarter ended June 30, 2022. Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, Inc., stated, “Through the ...
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RareStone Inc. Announces Strategic Collaboration with Tencent on a Service Ecosystem Focused on Rare Disease Patients in China
Holdings Limited (Tencent),based in Shenzhen,China,signed a strategic collaboration memorandum to develop a service ecosystem focused on rare disease patients in China. The combined effort will promote education of rare disease,focus social awareness on rare disease,and improve accessibility of medical information and services for rare disease by working together to provide innovative digital ...
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Comprehensive Gene Therapy Development Solutions to Further Boost Rare Disease Research
As several gene therapy approvals for rare disease treatment gain steam in 2023, a comprehensive set of gene therapy development solutions is released to pave richer avenues for rare disease research. This path-breaking move sees the integration of cutting-edge technology with state-of-art research facilities to amplify the possibilities of devising effective remedies for rare diseases. Rare ...
By Protheragen
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Lumos Pharma to Participate in the Cantor Rare Disease Symposium
Lumos Pharma, Inc. (NASDAQ:LUMO), a clinical-stage biopharmaceutical company focused on therapeutics for rare diseases, announced that John McKew, PhD, President and Chief Scientific Officer of Lumos Pharma, will participate in a panel discussion at the Cantor Rare Disease Symposium at the end of March 2022. Event: Cantor Rare Disease Symposium – March 28-30 Presentation: Panel ...
By Lumos Pharma
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Population Bio Recognizes World Rare Disease Day 2017 with a Commitment to Provide New Insights into Rare Diseases
Pioneering technology platform rapidly uncovers the genetic causes of unsolved diseases and redefines the understanding of rare and complex genetic diseases. Melville, NY, (February 28, 2017) Population Bio (PB) – a global leader in gene discovery announced, in support of World Rare Disease Day, its continued investment in its CNV Beacon® gene discovery platform which delivers new ...
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Healx pledges $25,000 to AKU Society patient registry campaign
Patient registries play a critical role in the development of treatments for rare diseases – 95% of which are still without an approved therapy today. Knowledge limitations around rare conditions are a huge barrier to treatment development and are compounded by the fact that rare disease patient populations are often small and geographically dispersed. Registries collect information ...
By Healx Ltd.
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Citrine medicine and Sinopharm group announce strategic partnership to broaden and accelerate access to rare disease drugs in china
Collaboration to focus on establishing supply chain and securing distribution channels for delivering rare disease therapies to patients across China BOSTON, Mass. and SHANGHAI, China, April 19, 2021 – Citrine Medicine, a China-based rare disease therapeutics company, and Sinopharm Group, a Chinese pharmaceutical company, today announced a strategic partnership to accelerate the ...
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3DBio Therapeutics Receives FDA Rare Pediatric Disease Designation for AuriNovo™ for Ear Reconstruction in Microtia Patients
3DBio Therapeutics, an emerging leader in the field of 3D-bioprinting for human therapeutic use, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation for AuriNovo™, an investigational combination product for reconstruction of the outer ear in patients with microtia. Microtia is a rare congenital disorder causing the outer ear ...
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Lumos Pharma to Present at the H.C. Wainwright BIOCONNECT Virtual Conference
Lumos Pharma, Inc. (NASDAQ:LUMO), a clinical-stage biopharmaceutical company focused on therapeutics for rare diseases, announced that the Company will present at the H.C. Wainwright Virtual BIOCONNECT Conference being held January 10-13, 2022. A webcast of the presentation will be available at 7:00AM ET on January 10th through the following link: ...
By Lumos Pharma
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Haymarket Medical Network Launches Rare Disease Advisor
Haymarket Medical Network today launched Rare Disease Advisor, a new digital resource for healthcare professionals (HCPs) to help them recognize, diagnose, refer and treat rare diseases. RareDiseaseAdvisor.com fills an information gap for HCPs by providing difficult to find information on rare diseases in one convenient place, enabling physicians and care teams with a comprehensive digital ...
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Healx launches AI-powered indication expansion partnership with Ono Pharmaceutical Co. Ltd.
Healx, the AI-powered, patient-inspired technology company pioneering the next generation of drug discovery, today announced an exciting partnership with Ono Pharmaceutical Co., Ltd., one of Japan’s largest pharmaceutical companies and a global leader in the field of R&D-orientated drug discovery. The collaboration will leverage Healx’s innovative indication expansion technology ...
By Healx Ltd.
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Chiesi Group and Holostem Terapie Avanzate announce transfer of Holoclar® from the Chiesi Group to Holostem
Parma and Modena, 16 June 2020 – Chiesi Farmaceutici, international research-focussed group (Chiesi Group), and Holostem Terapie Avanzate, top Italian biotech company entirely dedicated to the development, production, registration and distribution of medicinal products for advanced therapies, announce the signing of a contract for the transfer of the business branch of Holoclar®, an ...
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Capricor Therapeutics to Participate in the Cantor Fitzgerald Rare Orphan Disease Summit
Capricor Therapeutics (NASDAQ: CAPR) a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of a broad spectrum of diseases, announced today that Dr. Linda Marbán, CEO of Capricor, will participate in a panel discussion at the virtual Cantor Fitzgerald Rare Orphan Disease Summit on March 30, 2022. Please see ...
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AI promises a new future of drug discovery for rare diseases
Despite affecting over 400 million people around the globe, rare diseases have largely been overlooked by traditional drug discovery methods. This is because traditional model often prioritise disease areas where there is a greater understanding of the basic underlying biology and which, in turn, generally come from the larger patient populations associated with more common diseases. This is part ...
By Healx Ltd.
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CENTOGENE and Molecular Health Announce an Exclusive Collaboration With the Aim to Transform the Development of Orphan Drugs
The combination of real-life data sets in rare diseases with innovative big data/artificial intelligence/novel scientific computational knowledge will transform biomedical research, product development, and therapy. Real-life data and Innovative Bioinformatic Algorithms (RIBA project) will enable the creation of a powerful environment to accelerate, de-risk, and improve the development of new ...
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CENTOGENE Announces Appointment of Miguel Coego Rios as Chief Financial Officer
Centogene N.V. (Nasdaq: CNTG), the commercial-stage, essential biodata life science partner for rare and neurodegenerative diseases, today announced the appointment of Miguel Coego Rios as Managing Director and Chief Financial Officer of the Company, effective June 22, 2022. “As we continue to make progress on pivoting the organization to focus on Core Business execution, we are very ...
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BioMarin and Deep Genomics to Collaborate on Advancing Programs Identified Using Artificial Intelligence
BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) and Deep Genomics today announced that the companies have entered into a preclinical collaboration that will use Deep Genomics’ artificial intelligence drug discovery platform (The AI Workbench) to identify oligonucleotide drug candidates in four rare disease indications with high unmet need. Deep Genomics will receive an undisclosed upfront ...
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Infant Bacterial Therapeutics publishes Annual Report for 2021
Infant Bacterial Therapeutics AB (publ) publishes today the Annual Report for 2021. The report is available on IBT’s website ibtherapeutics.com under the section “Investors & Media – Financial Reports” and is available in printed form for those that have requested. A copy can be requested by contacting IBT’s office via info@ibtherapeutics.com. About Infant ...
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