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Rare Diseases Chard And The Chinese Red Cross Foundation Crcf Jointly Initiated The Rare Disease Articles & Analysis

237 news found

Huateng Pharma's mPEG-pALD Receives FDA DMF Filing Approval

Huateng Pharma's mPEG-pALD Receives FDA DMF Filing Approval

Changsha, China – Hunan Huateng Pharmaceutical Co., Ltd. (Huateng Pharma), a global leader in the development and production of high-quality PEG derivatives, is pleased to announce that its mPEG-pALD (20K) has successfully completed its Drug Master File (DMF) filing with the U.S. Food and Drug Administration (FDA), under DMF No. 040600. This achievement highlights Huateng Pharma’s ...

ByHunan Huateng Pharmaceutical Co. Ltd.


Diving Deeper in Rare Disease Research: Protheragen Unveils Diagnostic Development Services

Diving Deeper in Rare Disease Research: Protheragen Unveils Diagnostic Development Services

Protheragen, a dedicated preclinical research service provider specializing in rare diseases, announces its comprehensive diagnostic development services aimed at empowering researchers in the realm of rare disease research and drug development. These diagnostic services are designed to support the scientific community in identifying, understanding, and ultimately treating rare diseases that ...

ByProtheragen


Protheragen Announces Vaccine Development Services Targeting Rare Infectious Diseases

Protheragen Announces Vaccine Development Services Targeting Rare Infectious Diseases

Protheragen is proud to release the launch of its comprehensive vaccine development solutions tailored to advance research and development in the field of infectious diseases. This strategic initiative aims to accelerate the creation of effective vaccines to combat emerging and re-emerging infectious threats. Infectious diseases are disorders caused by organisms — such as bacteria, ...

ByProtheragen


SOPHiA GENETICS and Genomenon Collaborate to Streamline Genetic Research

SOPHiA GENETICS and Genomenon Collaborate to Streamline Genetic Research

SOPHiA GENETICS (Nasdaq: SOPH), a cloud-native healthcare technology leader in data-driven medicine, today announced the next step in the partnership between the Company and Genomenon, a leading genomic intelligence company, to support better, data-driven outcomes in the rare disease and oncology fields. The two companies have expanded the integration of Genomenon’s Mastermind® Genomic ...

BySOPHiA Genetics


H5N1 Antibodies and Antigens for Research Applications

H5N1 Antibodies and Antigens for Research Applications

Creative Diagnostics, a leading manufacturer and supplier of antibodies, antigens and assay kits, is excited to announce the launch of its new line of H5N1 Antibodies and Antigens to support cutting-edge research on this highly pathogenic influenza A virus subtype. These innovative products offer researchers and scientists invaluable tools for studying and combating the H5N1 influenza virus. ...

ByCreative Diagnostics


Biotech Stock News Bite - Longeveron Inc. (NASDAQ: LGVN) Stock Soars on Clinical Pipeline and 2024 Key Priorities and Goals

Biotech Stock News Bite - Longeveron Inc. (NASDAQ: LGVN) Stock Soars on Clinical Pipeline and 2024 Key Priorities and Goals

Investorideas.com (www.investorideas.com) Biotech Stock News Bites - Longeveron Inc. (NASDAQ: LGVN), a clinical stage biotechnology company developing regenerative medicines is one of NASDAQ's top gainers today and the stock is trending on Yahoo Finance, currently trading at $3.8955, up $1.6955 or 77.0682%. The stock had a morning high of $5.47 on volume of over 36 Million/. The Company's CEO, ...

ByInvestorideas.com


Comprehensive Gene Therapy Development Solutions to Further Boost Rare Disease Research

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 ...

ByProtheragen


Clinical Trial Managers Need to Know These Skills for the Future

Clinical Trial Managers Need to Know These Skills for the Future

The Covid-19 epidemic has changed the world in many ways, and many clinical trials have been done to research more about it. According to the World Health Organization (WHO), 12,969 clinical trials were done in America in 2021. Even in pre-pandemic times, clinical trials have always been an ongoing process. Clinical trial managers work on cutting-edge studies and contribute to ...

ByBrio Group


SeqOne Genomics and the French Thrombotic MicroAngiopathies National reference center (CNR-MAT) pioneer the use of Oxford Nanopore sequencing technology to improve patient outcomes in kidney disease while reducing turnaround times

SeqOne Genomics and the French Thrombotic MicroAngiopathies National reference center (CNR-MAT) pioneer the use of Oxford Nanopore sequencing technology to improve patient outcomes in kidney disease while reducing turnaround times

SeqOne today announced the results of a long-standing research collaboration with Pr. Laurent Mesnard, Co-Director of the French National center for Thrombotic MicroAngiopathies (CNR-MAT), aimed at improving the diagnosis and management of patients suspected of suffering from thrombotic microangiopathies and atypical Hemolytic syndrome (aHUS). The program involved using Oxford Nanopore’s ...

BySeqOne S.A.S.


FDA U.S. Food & Drug Administration – Action Plan for Rare Neurodegenerative Diseases including Amyotrophic Lateral Sclerosis

FDA U.S. Food & Drug Administration – Action Plan for Rare Neurodegenerative Diseases including Amyotrophic Lateral Sclerosis

A five-year action plan developed to meet requirements under Section 4 of the Accelerating Access to Critical Therapies for ALS Act. ...

ByVirpax Pharmaceuticals


Roswell Biotechnologies Forms Scientific Advisory Board

Roswell Biotechnologies Forms Scientific Advisory Board

Roswell Biotechnologies, Inc., the molecular electronics company, announced today the formation of its Scientific Advisory Board (SAB). Comprised of industry and academic leaders in science and technology, the new SAB will be instrumental in the final stages of development and commercialization of the company's molecular electronics chip, a fully scalable universal biosensor capable of seeing ...

ByRoswell Biotechnologies Inc


Nautilus Biotechnology Partners with the Translational Genomics Research Institute (TGen) to Investigate Applications of Single-Molecule Proteomic Analysis in Diffuse Intrinsic Pontine Glioma (DIPG)

Nautilus Biotechnology Partners with the Translational Genomics Research Institute (TGen) to Investigate Applications of Single-Molecule Proteomic Analysis in Diffuse Intrinsic Pontine Glioma (DIPG)

Nautilus Biotechnology, Inc. (NASDAQ: NAUT; or “Nautilus”), a company pioneering a single-molecule protein analysis platform, and the Translational Genomics Research Institute (TGen), part of City of Hope, today announced a partnership to explore the utility of the Nautilus platform by studying specific protein targets in diffuse intrinsic pontine glioma (DIPG), a rare and often fatal ...

ByNautilus Biotechnology


Oligomerix Presents Data on Lead Program at Clinical Trials on Alzheimer’s Disease (CTAD) Conference and Society for Neuroscience Meeting

Oligomerix Presents Data on Lead Program at Clinical Trials on Alzheimer’s Disease (CTAD) Conference and Society for Neuroscience Meeting

Oligomerix, Inc., a privately held company pioneering the development of small molecule therapeutics targeting tau for Alzheimer’s and rare neurodegenerative diseases, announced that the Company presented data from a therapeutic study in an animal model of its lead product candidate OLX-07010 at the 14th Annual Clinical Trials on Alzheimer's Disease (CTAD) conference and at the Society for ...

ByOligomerix, Inc.


NextPoint Therapeutics Announces $80 Million Series B Financing co-led by Leaps by Bayer and Sanofi Ventures to Advance Novel Immuno-Oncology Programs

NextPoint Therapeutics Announces $80 Million Series B Financing co-led by Leaps by Bayer and Sanofi Ventures to Advance Novel Immuno-Oncology Programs

NextPoint Therapeutics, a biotechnology company developing a new world of precision immuno-oncology, announced today that it raised $80 million in Series B financing co-led by Leaps by Bayer, the impact investment arm of Bayer AG, and Sanofi Ventures, the strategic venture capital arm for Sanofi. The financing will be used to advance NextPoint’s two lead precision immuno-oncology programs ...

ByBayer AG


Eagle Pharmaceuticals Provides Business Update and Guidance for 2023

Eagle Pharmaceuticals Provides Business Update and Guidance for 2023

Eagle Pharmaceuticals, Inc. (Nasdaq: EGRX) (“Eagle” or the “Company”) today provided a business update and guidance for 2023. Highlights: During the 12 months ended September 30, 2022, Eagle recorded net income of $21.3 million or $1.63 per diluted share and adjusted EBITDA of $125.6 million and non-GAAP earnings per diluted share of $7.54, a significant increase from ...

ByEagle Pharmaceuticals, Inc.


BRIM Biotechnology receives FDA Orphan Drug Designation for BRM424 to treat neurotrophic keratitis

BRIM Biotechnology receives FDA Orphan Drug Designation for BRM424 to treat neurotrophic keratitis

BRIM Biotechnology, Inc. (“BRIM,” TPEx 6885) is pleased to announce that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for BRM424 in the treatment of neurotrophic keratitis (NK), a rare degenerative eye disease which causes very severe cornea damage and can lead to loss of sight. Dr. Haishan Jang, Chairwoman and CEO of BRIM Biotechnology, ...

ByBRIM Biotechnology, Inc.


LISCure`s Phase 2 Drug Candidate Granted Orphan Drug Designation (ODD) from US FDA

LISCure`s Phase 2 Drug Candidate Granted Orphan Drug Designation (ODD) from US FDA

LB-P8, a novel drug being developed by LISCure Biosciences, Inc. (LISCure) as a treatment for Primary Sclerosing Cholangitis (PSC), has been designated as an orphan drug by the US FDA. On December 1, 2022, the US FDA notified LISCure of the orphan drug designation of LB-P8, LISCure's microbiome novel drug, as treatment of PSC. LB-P8 is, to the best of company's knowledge, the first drug to be ...

ByLISCure Biosciences Inc.


Lumos Pharma to Present at the H.C. Wainwright BIOCONNECT Virtual Conference

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: ...

ByLumos Pharma


CY6463 administration is linked to improvements in Alzheimer’s disease-relevant biomarkers, as revealed by eXplainable AI-driven analysis of multiple Phase 1 clinical trials ?

CY6463 administration is linked to improvements in Alzheimer’s disease-relevant biomarkers, as revealed by eXplainable AI-driven analysis of multiple Phase 1 clinical trials ?

Phase 1 studies show improvement in cognitive factors, consistent with therapeutic potential of CY6463 to improve key features in neurological diseases CY6463 favorable tolerability and safety profile was confirmed Ariana Pharma, a leading Artificial Intelligence (AI) drug development company, in collaboration with Cyclerion Therapeutics, presented today safety and pharmacodynamic results ...

ByAriana Pharma


Eikonoklastes Therapeutics and Forge Biologics Announce Viral Vector Contract Development and AAV Manufacturing Partnership

Eikonoklastes Therapeutics and Forge Biologics Announce Viral Vector Contract Development and AAV Manufacturing Partnership

Eikonoklastes Therapeutics, a preclinical biotech company developing treatments for today’s most challenging diseases, and Forge Biologics, a gene therapy-focused contract development and manufacturing organization, today announced a manufacturing partnership that will advance Eikonoklastes’ AAV-based gene therapy, ET-101, into clinical trials for the treatment of patients with ...

ByForge Biologics, Inc.

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