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Rare Diseases

Showing 15 posts of 33 posts found.

Results from Novadip phase 1b/2a trial reveal 88% efficacy

January 28, 2025
Clinical 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 …

News

J&J seek approval for first drug to treat high-risk smoldering myeloma

November 13, 2024
Research 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 …

News
cancer_drip_2

Chiesi announces phase 3 results from enzyme-replacement study

October 29, 2024
Research 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 …

News

VALIANT phase 3 trial results highlight potential treatment for rare kidney diseases

October 25, 2024
Nephrology, 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 …

News

Chiesi Global Rare Diseases Launches New Research Grant Initiative to Support Research Excellence in Lysosomal Storage Disorders

September 9, 2024
Research 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 …

News
Press release

Quince Therapeutics gains FDA Fast Track Designation for EryDex System

June 4, 2024
Medical 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 …

News

Rare disease clinical trials: the urgent need for patients to be heard

May 22, 2024
Alexion, 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 …

Feature
genes

Shionogi and Maze Therapeutics announce worldwide exclusive licensing agreement for novel Pompe disease candidate

May 10, 2024
Sales 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 …

News

AstraZeneca to acquire Amolyt Pharma for up to $1.05bn

March 14, 2024
Business 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 …

News

Sanofi to acquire Inhibrx for approximately $1.7bn

January 23, 2024
Business 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 …

News

Health Canada approves Ipsen and Medision Pharma’s Bylvay for pruritus treatment

November 14, 2023
Medical 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 …

News

FDA approves first ever treatment for rare CHAPLE disease

August 21, 2023
Research 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 …

News

Amicus Therapeutics’ Pompe disease treatment approved for use in UK

August 17, 2023
Medical 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 …

News
genes

AstraZeneca’s Alexion partners with Pfizer for rare disease therapies

July 28, 2023
Research 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 …

News

Santhera and Catalyst Pharmaceuticals sign $231m license and collaboration deal

June 20, 2023
Medical 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 …

News
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