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AzurBio Pharma Launches to Fast-Track European Market Entry for Biopharma Companies Pharmasources
October 14, 2024
AzurBio Pharma is announcing its official launch.
FDA joins Bespoke Gene Therapy Consortium (BGTC) for rare diseases EuropeanPharmaceuticalReview
October 29, 2021
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.
Fulcrum Therapeutics Announces Proposed Public Offering of Common Stock firstwordpharma
August 11, 2021
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.
Rallybio Corporation Announces Closing of Initial Public Offering and Full Exercise of the Underwriters’ Option to Purchase Additional Shares firstwordpharma
August 03, 2021
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.
Low awareness of rare diseases increases morbidity and potential mortality pharmaceutical-technology
June 02, 2021
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.
US FDA awards six clinical trials research grants to develop medical products for rare diseases expresspharma
October 10, 2020
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.
WuXi AppTec to Launch a Special Webinar on Collaborations that Transform on July 16 | Advances in Rare Diseases: Hemophilia prnasia
July 09, 2020
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.
Takeda partners with Codexis on gene therapies for rare diseases pharmaceutical-technology
March 25, 2020
Takeda Pharmaceutical has signed a strategic collaboration and licence agreement with biotherapeutics developer Codexis to research and create gene therapies for rare disorders.
PUMCH brings hope in rare diseases and Scoliosis patients biospectrumasia
August 29, 2019
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
Biologics or small molecule drugs for rare disease treatment? It is a question PharmaSources/Miling
July 31, 2019
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.

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