Rare Diseases
Results from Novadip phase 1b/2a trial reveal 88% efficacy
January 28, 2025Clinical Research Congenital Pseudoarthritis of the Tibia, Rare Diseases, clinical trials, efficacy, paediatric, rare diseases
Novadip Biosciences, a clinical stage biotechnology company with a specialty in regenerative medicine, has announced the results of its 12-month …
J&J seek approval for first drug to treat high-risk smoldering myeloma
November 13, 2024Research and Development FDA, J&J, Rare Diseases, bone marrow
Earlier in November, Johnson & Johnson innovative medicine have applied to the US Food and Drug Agency (FDA) and the …
Chiesi announces phase 3 results from enzyme-replacement study
October 29, 2024Research and Development Internal Medicine, Rare Diseases, chiesi Global rare diseases, clinical trials, pharma, rare diseases
Chiesi Global Rare Diseases have announced the results from their BRIGHT trial, which evaluates the efficacy of enzyme-replacement therapy drug …
VALIANT phase 3 trial results highlight potential treatment for rare kidney diseases
October 25, 2024Nephrology, Rare Diseases, rare kidney disease, trial
SOBI and Apellis Pharmaceuticals have announced the results from the VALIANT trial, which highlight that systemic pegcetacoplan treatment could benefit …
Chiesi Global Rare Diseases Launches New Research Grant Initiative to Support Research Excellence in Lysosomal Storage Disorders
September 9, 2024Research and Development Rare Diseases
9 September 2024, –London, UK –Applications for the independently assessed, expert-led Research Grant Initiative, Find For Rare, are open from …
Quince Therapeutics gains FDA Fast Track Designation for EryDex System
June 4, 2024Medical Communications EryDex, FDA, Quince Therapeutics, Rare Diseases, fast track designation
Quince Therapeutics has announced that the US Food and Drug Administration (FDA) has granted Fast Track Designation for its EryDex …
Rare disease clinical trials: the urgent need for patients to be heard
May 22, 2024Alexion, Pharmafocus, Rare Diseases, clinical trials, feature
Ahead of Clinical Trials Day, Soraya Bekkali, from Alexion, AstraZeneca Rare Disease, highlights the urgent need to elevate patients’ voices …
Shionogi and Maze Therapeutics announce worldwide exclusive licensing agreement for novel Pompe disease candidate
May 10, 2024Sales and Marketing Pompe disease, Rare Diseases, Shionogi, licensing agreement, maze therapeutics
Japanese pharmaceutical company Shionogi and US-based Maze Therapeutics have finalised an exclusive worldwide license agreement for MZE001, a novel therapeutic …
AstraZeneca to acquire Amolyt Pharma for up to $1.05bn
March 14, 2024Business Services Amolyt Pharma, AstraZeneca, Rare Diseases, acquisition, rare diseases
AstraZeneca has announced that it has entered into a definitive agreement under which it will acquire Amolyt Pharma for up …
Sanofi to acquire Inhibrx for approximately $1.7bn
January 23, 2024Business Services Inhibrx, Rare Diseases, Sanofi, acquisition
Sanofi and Inhibrx, Inc have announced that they have entered into a definitive agreement for Sanofi to acquire Inhibrx following …
Health Canada approves Ipsen and Medision Pharma’s Bylvay for pruritus treatment
November 14, 2023Medical Communications Health Canada, Medision Pharma, Rare Diseases, ipsen, pruritis
Ipsen and Medision Pharma have announced that Health Canada has approved Bylvay (odevixibat) for the treatment of pruritis in patients …
FDA approves first ever treatment for rare CHAPLE disease
August 21, 2023Research and Development CHAPLE disease, FDA, Rare Diseases, rare disease
The US Food and Drug Administration (FDA) has approved Regeneron’s Veopoz (pozelimab), a new treatment for CHAPLE disease for both …
Amicus Therapeutics’ Pompe disease treatment approved for use in UK
August 17, 2023Medical Communications Amicus Therapeutics, MHRA, NICE, Pompe disease, Rare Diseases
Amicus Therapeutics has announced that the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK has granted marketing authorisation …
AstraZeneca’s Alexion partners with Pfizer for rare disease therapies
July 28, 2023Research and Development Alexion, AstraZeneca, Pfizer, Rare Diseases, genomics, rare disease
Alexion, AstraZeneca’s Rare Disease division, has announced that it has entered a definitive purchase and licence agreement with Pfizer. The …
Santhera and Catalyst Pharmaceuticals sign $231m license and collaboration deal
June 20, 2023Medical Communications Catalyst Pharmaceuticals, Rare Diseases, Vamorolone, licence and collaboration, santhera
Swiss specialty pharmaceutical company Santhera and US-based biopharmaceutical company Catalyst Pharmaceuticals have announced that they have signed an exclusive license …
