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Rare Disease Research Articles & Analysis

8 news found

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

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

At Huateng Pharma, we are committed to fully supporting our customers' research and clinical needs with high-quality, compliant PEG products. We look forward to continuing our collaborations and partnerships, contributing to advancements in drug development and improving patient outcomes." mPEG-pALD (20K) plays a vital role in various applications, including the development of ...

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. ...

ByProtheragen


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 ...

ByProtheragen


HemoShear Develops New Human Tissue-Based Model of Rare Liver Disease Under Research Collaboration with Takeda

HemoShear Develops New Human Tissue-Based Model of Rare Liver Disease Under Research Collaboration with Takeda

HemoShear Therapeutics, Inc., a privately held clinical stage biotechnology company, has received a milestone payment under its October 2020 research collaboration and service agreement with Takeda. The milestone acknowledges development of a human tissue-based model of an undisclosed rare liver disease using the company’s REVEAL-Tx™ ...

ByHemoShear Therapeutics, Inc.


CENTOGENE Announces Appointment of Miguel Coego Rios as Chief Financial Officer

CENTOGENE Announces Appointment of Miguel Coego Rios as Chief Financial Officer

(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. ...

ByCENTOGENE N.V.


The China Alliance for Rare Diseases (CHARD) and the Chinese Red Cross Foundation (CRCF) jointly initiated the `Rare Disease Mutual Assistance Fund`.

The China Alliance for Rare Diseases (CHARD) and the Chinese Red Cross Foundation (CRCF) jointly initiated the `Rare Disease Mutual Assistance Fund`.

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" ...

ByRareStone Group


Pfizer and Sangamo Announce Updated Phase 1/2 Results Showing Sustained Bleeding Control in Highest Dose Cohort Through Two Years Following Hemophilia A Gene Therapy

Pfizer and Sangamo Announce Updated Phase 1/2 Results Showing Sustained Bleeding Control in Highest Dose Cohort Through Two Years Following Hemophilia A Gene Therapy

The Pfizer focus on rare disease builds on more than two decades of experience, a dedicated research unit focusing on rare disease, and a global portfolio of multiple medicines within a number of disease areas of focus, including rare hematologic, neurologic, cardiac and ...

BySangamo Therapeutics


NanoTemper Technologies teams up with PharmAI to help researchers spot game-changing drug candidates from millions of possibilities

NanoTemper Technologies teams up with PharmAI to help researchers spot game-changing drug candidates from millions of possibilities

Together, they will zero in on one of the biggest roadblocks to quick drug development: Researchers have to comb through large amounts of data to find the right drug candidates. This process is an innovation killer, making it difficult for small laboratories to do drug research and even discouraging large pharmaceutical companies from going after ...

ByPharmAI GmbH

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