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AzurBio Pharma is announcing its official launch.
October 14, 2024
by Pharmasources
The US Food and Drug Administration (FDA), the National Institutes of Health (NIH), 10 pharmaceutical companies and five non-profit organisations have partnered to accelerate development of gene therapies for rare diseases.
October 29, 2021
by EuropeanPharmaceuticalReview
Fulcrum Therapeutics, Inc. (Nasdaq: FULC) today announced that it has commenced an underwritten public offering of $100 million of shares of its common stock. All of the shares are being offered by Fulcrum.
August 11, 2021
by firstwordpharma
Rallybio Corporation today announced the closing of its initial public offering of 7,130,000 shares of common stock, including the exercise in full by the underwriters of their option to purchase up to 930,000 additional shares of common stock.
August 3, 2021
by firstwordpharma
Orphan or rare diseases are conditions that affect a small percentage of the population in a given country. The widely accepted definition in the US is a disease that affects fewer than 200,000 people nationwide.
June 2, 2021
by pharmaceutical-technology
FDA received 47 clinical trial grant applications that were reviewed and evaluated for scientific and technical merit by more than 90 rare disease and clinical trial experts.
October 10, 2020
by expresspharma
As an enabler of the biotech and medical device industry, WuXi AppTec shares the conviction that we must work collaboratively to solve industry-wide problems and bring transformational medicines to people living with rare diseases.
July 9, 2020
by prnasia
Takeda Pharmaceutical has signed a strategic collaboration and licence agreement with biotherapeutics developer Codexis to research and create gene therapies for rare disorders.
March 25, 2020
by pharmaceutical-technology
The 2018 PUMCH Medical Research award winner has achieved through surgery teams in the department of Orthopaedics by diagnosing 33 rare diseases involving 7 major systems in patients with scoliosis based on routine clinical diagnostic techniques, molecula
August 29, 2019
by biospectrumasia
To both patients and pharmaceutical practitioners, it is a question as to whether to choose biologics or small molecule drugs for rare disease treatment, since the prices are sky-high.
July 31, 2019
by PharmaSources/Miling
One key way we’ve addressed these opportunities is by providing funding for rare disease natural history ……
March 23, 2019
by firstwordpharma
Clarivate Analytics announced today the launch of its annual "Cortellis Drugs to Watch" report.
March 21, 2019
by en-cphi.cn
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