Sanofi Reports P-III (LEAP2MONO) Trial Data on Venglustat in Type 3 Gaucher Disease

Shots: Sanofi has reported P-III (LEAP2MONO) trial data assessing venglustat (QD, PO) vs enzyme replacement therapy (IV, Q2W) in 43 pts (≥12yrs.) with GD3 Trial met its 1EP, improving neurological symptoms measured by modified SARA total score & RBANS at Wk. 52, & achieved 3 of 4 key 2EPs, incl. reductions in spleen & liver…

ByRidhi RastogiFebruary 3, 2026

NEWS

BioMarin to Acquire Amicus Therapeutics for ~$4.8B

Shots: BioMarin has entered into a definitive agreement to acquire Amicus, strengthening BioMarin's commercial portfolio with Galafold (migalastat) for Fabry Disease & Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) for Pompe Disease As per the deal, BioMarin will acquire Amicus for $14.5/share in an all-cash transaction for a total equity value of ~$4.8B; closing is expected in Q2’26 Amicus also…

ByRidhi RastogiDecember 22, 2025

INSIGHTS+

Disease of the Month – Fabry Disease

Shots: Fabry disease is a rare inherited neurological disorder that affects the body's ability to break down lipids into smaller components In this reprise of our Disease of the Month report, we bring an illuminating account of Fabry Disease with deep dive analysis of epidemiology, market size, disease management, available therapies, patient advocacy groups, and…

BySenior EditorJune 12, 2024

VIEWPOINTS

Bettina Cockroft, SVP & CMO at Sangamo Therapeutics Shares Insights from the Positive P-I/II Data for Fabry Disease Treatment

Shots: Bettina gave the details of the study design and shared the preliminary results from the P-I/II STAAR clinical study evaluating ST-920. The data were presented in an oral presentation at the 29th Congress of the European Society of Gene & Cell Therapy 2022 She also talked about enzyme replacement therapy (ERT) which is currently used for the…

BySenior EditorApril 24, 2023

THOUGHTSPOT

Getting on with Rare Disease Therapeutic Options (Orphan Disease Therapies)

Shots: With the healthcare sector moving ahead with its technological advances, there is still an unexplored section in therapeutics that requires much more attention. Through innovations, life science companies have evolved to make breakthrough discoveries in many focus areas, but when it comes to the rare disease segment, all our endeavors seem to be bleak…

BySaurabh ChaubeyMarch 14, 2023

Sanofi Reports P-III (LEAP2MONO) Trial Data on Venglustat in Type 3 Gaucher Disease

BioMarin to Acquire Amicus Therapeutics for ~$4.8B

Disease of the Month – Fabry Disease

Bettina Cockroft, SVP & CMO at Sangamo Therapeutics Shares Insights from the Positive P-I/II Data for Fabry Disease Treatment

Getting on with Rare Disease Therapeutic Options (Orphan Disease Therapies)

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