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rare disease

This page shows the latest rare disease news and features for those working in and with pharma, biotech and healthcare.

FDA expecting boom in cell and gene therapies

FDA expecting boom in cell and gene therapies

The last couple of years have already seen the approval of the first gene therapy in the US – Spark Therapeutics’ Luxturna for a rare disease – as well as new cell therapies ... its course by altering the expression of a protein or enzyme believed

Latest news

  • Orphan drugs dominate FDA's record-breaking year Orphan drugs dominate FDA's record-breaking year

    Well over half of the new approvals were for rare diseases, helped by an FDA drive to eliminate a backlog in orphan drug reviews that kicked off with the Orphan Drug ... That included Alexion’s Ultomiris (ravulizumab) for paroxysmal nocturnal

  • Afinitor and Uptravi gain funding from NHS England Afinitor and Uptravi gain funding from NHS England

    Rare disease treatments navigate tricky market access route. Novartis’ Afinitor in Tuberous Sclerosis Complex (TSC)-related epilepsy patients and Actelion’s pulmonary arterial hypertension (PAH) treatment Uptravi (selexipag) have gained funding

  • Chugai gains FDA breakthrough status for rare CNS treatment Chugai gains FDA breakthrough status for rare CNS treatment

    We continue our efforts to hopefully bring satralizumab as a new treatment option as soon as possible to people living with this devastating disease with no approved drugs.”. ... NMOSD is a rare, lifelong, and debilitating autoimmune disease of the

  • Axovant drops last ‘legacy’ drug to focus on gene therapies Axovant drops last ‘legacy’ drug to focus on gene therapies

    licensed rights to a gene therapy for Parkinson’s disease (PD) from UK biotech Oxford BioMedica for $30m upfront. ... That was followed swiftly by a $10m upfront deal with Benitec Biopharma for rights to a preclinical stage gene therapy for rare

  • Genomics England reaches 100,000th genome sequencing milestone Genomics England reaches 100,000th genome sequencing milestone

    those with rare inherited diseases and cancer. ... Due to the personalised nature of genome sequencing, one in four patients with a rare disease received a diagnosis for the very first time.

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Latest Intelligence

  • Relearning launch: Four key dynamics for pharma launches in 2019 Relearning launch: Four key dynamics for pharma launches in 2019

    Being ready for those rare, high-potential launches is the no. 1 imperative in biopharmaceutical innovation today. ... Gene and cell therapies, novel approaches to rare disease and drug replacements, like digital therapies, are about to crowd the market,

  • Alnylam’s big moment: bringing groundbreaking RNAi drugs to Europe Alnylam’s big moment: bringing groundbreaking RNAi drugs to Europe

    Heggie has lots of experience in the rare disease field, having served in senior roles at Shire, as a board member at Sobi and as CEO of German rare disease biotech ... Diagnosis. As with many new rare disease therapies, one of the biggest challenges for

  • The European Medicines Agency: PRIME’d for access? The European Medicines Agency: PRIME’d for access?

    RG6042 (formerly named IONIS-HTTRx) is in development for Huntington’s disease. ... It’s hoped it could be a big step in treatment for the genetically-inherited and rare wasting disease.

  • Bluebird Bio: on the cusp of a gene therapy revolution Bluebird Bio: on the cusp of a gene therapy revolution

    pancreatitis. Then GSK’s Strimvelis followed in 2016 for a very rare disease called ADA-SCID (Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency). ... However this small molecule drug promises more modest treatment benefits, and

  • Accelerating diagnosis in rare disease Accelerating diagnosis in rare disease

    Biopharmaceutical companies can drive effective partnerships and improve outcomes for all stakeholders and patients in the area of rare disease. ... Timely diagnosis in rare disease is one of the fundamental unmet needs for patients, healthcare

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Latest appointments

  • Ipsen hires general manager, Laura Hamill joins Gilead and Orchard expands leadership team Ipsen hires general manager, Laura Hamill joins Gilead and Orchard expands leadership team

    expertise as we shape our business for future growth with an unrelenting commitment to enhancing the lives of patients living with rare disease.

  • BMS appoints new commercial leader, Davies joins Eisai and more BMS appoints new commercial leader, Davies joins Eisai and more

    Sobi appoints head of technical operations. Rare disease specialist company Sobi has appointed Anne Marie de Jonge Schuermans to front its new technical operations organisation. ... Commenting on her appointment, Schuermans said: “Sobi has unique

  • Abeona Therapeutics names CEO Abeona Therapeutics names CEO

    Carsten Thiel joins the biopharma from Alexion. Rare genetic disease focused biopharma Abeona Therapeutics has named its chief executive officer in the form of Carsten Thiel. ... Steven Rouhandeh, executive chairman of Abeona, said: “As CEO, Carsten

  • Therachon poaches Shire’s Aled Williams Therachon poaches Shire’s Aled Williams

    Williams has more than 25 years’ experience in healthcare and pharmaceuticals and most recently he served as vice president and global strategy head for rare gastrointestinal diseases at Shire. ... Commenting on the recent appointment, Luca Santarelli,

  • Open Health creates new role Open Health creates new role

    In order to strengthen their experience and expertise, healthcare communications and market access group Open Health has appointed Gavin Jones into its newly formed role of director of rare disease. ... Commenting on his appointments, Sandy Roydon, chief

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