Sanofi records win and loss with rare disease drug in Phase III trials
Sanofi’s rare disease drug venglustat has met the primary endpoint in a Phase III trial in Gaucher disease but missed…
Genetic Disorders
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Sarepta to investigate immunosuppressant Elevidys combo to avoid fatal AEs
Sarepta Therapeutics will be investigating the potential of using an immunosuppressant both before and after treatment with Elevidys (delandistrogene moxeparvovec-rokl)…
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Harmony’s cannabidiol drug flops in pivotal Fragile X Syndrome trial
Harmony Biosciences’ transdermal cannabidiol ZYN002 suffered a setback as a Phase III trial failed to meet the primary endpoint in…
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Avidity touts disease reversal potential of DMD therapy
Avidity Biosciences’ Duchenne muscular dystrophy (DMD) therapy, del-zota, demonstrated reversal of disease progression in a Phase I/II and open-label extension…
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Pfizer’s sickle cell disease candidate fails Phase III trial
Pfizer is mulling its options after its investigational sickle cell disease drug failed to meet the primary endpoint in a…
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Taiho’s DMD therapy misses the mark in Phase III trial
Taiho Pharmaceutical’s investigational Duchenne muscular dystrophy (DMD) therapy, pizuglanstat, has shown no benefit in a Phase III trial. The REACH-DMD…
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Scholar Rock SMA drug shows muscle function benefit
Scholar Rock’s spinal muscular atrophy (SMA) therapy has shown benefit in muscle function in children under 12 in a pivotal…
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PepGen pauses Phase II DMD trial to review safety data
PepGen is pausing the initiation of a new Phase II Duchenne muscular dystrophy (DMD) study as it awaits data from…
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Why pharma is still far from a treatment for galactosemia
Nearly three months after an experimental treatment for classic galactosemia was rejected by the US Food and Drug Administration (FDA),…
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Korro secures approvals in Australia to commence AATD trial of KRRO-110
Korro Bio has secured approvals in Australia to commence the two-part Phase I/IIa REWRITE trial of its investigational product, KRRO-110,…
