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8th July 2024
Myrtelle has announced that their gene therapy, rAAV-Olig001-ASPA for the treatment of Canavan disease has been chosen for the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program.
28th June 2024
We carried out a survey with the assistance of the principal investigators in the leriglitazone trials to treat cerebral Adrenoleukodystrophy (ALD). The impact report is available to view.
27th June 2024
The June edition of our monthly news round-up has been sent out today. It includes charity news, our community and ways to get involved.
26th June 2024
Spur Therapeutics have announced their acquisition of SwanBio Therapeutics, adding Phase 1/2 program for Adrenomyeloneuropathy (AMN) to its clinical-stage pipeline and strengthening CNS expertise.
26th June 2024
New clinical sites open in US and Germany for Ionis ORBIT Phase 1 clinical trial of an investigational drug, ION356, in people with Pelizaeus-Merzbacher Disease (PMD)
26th June 2024
SHCA has published a new report on reducing delays to a rare disease diagnosis, analysing the progress made over the past year against existing government actions and commitments.
21st June 2024
Passage Bio has received FDA Clearance of IND Application for PBML04 for Treatment of Metachromatic Leukodystrophy. Read the press release to find out more.
21st June 2024
Calico Life Sciences Announces that Fosigotifator (ABBV-CLS-7262) for Vanishing White Matter Disease has been Selected for the FDA START Pilot Program.
18th June 2024
Have you been negatively affected by the recent DfE home to school transport guidance? Share your experiences by completing a survey by Cerebra, IPSEA, SOSSEN and Contact.
18th June 2024
UMass Chan has received $2.2 million donation from the Riaan Research Initiative to fund gene therapy for Cockayne syndrome.
18th June 2024
School transport is consistently in the top 10 issues on Contact’s education helpline – Contact’s have announced a new school transport project and Channel 4 News have investigated the post-16 transport issue in a film.
